Fidest – Agenzia giornalistica/press agency

Quotidiano di informazione – Anno 33 n° 338

Posts Tagged ‘fda’

ViiV Healthcare receives complete response letter from US FDA

Posted by fidest press agency su martedì, 24 dicembre 2019

ViiV Healthcare, the global specialist HIV company majority owned by GSK, with Pfizer Inc. and Shionogi Limited as shareholders, received a complete response letter (CRL) from the US Food and Drug Administration (FDA) regarding its application for cabotegravir and rilpivirine long-acting regimen for treatment of HIV-1 infection in virologically suppressed adults.The reasons given in the CRL relate to Chemistry Manufacturing and Controls (CMC). There have been no reported safety issues related to CMC and there is no change to the safety profile of the products used in clinical trials to date. ViiV Healthcare will work closely with the FDA to determine the appropriate next steps for this New Drug Application. Cabotegravir is an integrase strand transfer inhibitor developed by ViiV Healthcare and rilpivirine is a non-nucleoside reverse transcriptase inhibitor developed by Janssen Sciences Ireland UC. The cabotegravir and rilpivirine long-acting regimen is an investigational product and not approved anywhere in the world.

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Elligo Receives FDA Grant to Study Access of Real-World Data From Electronic Health Records

Posted by fidest press agency su venerdì, 4 ottobre 2019

Elligo Health Research, a healthcare-enabling research organization, has received a grant from the U.S. Food and Drug Administration (FDA) for a follow-on project to extend the value of common data harmonization in the generation of clinical evidence from real-world data (RWD) to support regulatory use. This grant will enable Elligo to explore methodology and use cases to further goals of the 21st Century Cures Act related to facilitating data sharing. Achievements from the first phase of this project include mappings of multiple data models (OMOP, PCORnet, i2b2, and Sentinel) to the BRIDG Model (an HL7, CDISC, and ISO standard), a reference data model and terminology bindings. These products, which have been catalogued by the National Cancer Institute, will be leveraged in the new project, along with HL7’s FHIR standard. “This project has been challenging, but educational and rewarding for us as an institution participating in multiple research networks that currently request de-identified data in as many different formats,” said Samuel Volchenboum, MD, PhD, associate chief research informatics officer of the University of Chicago’s Center for Research Informatics. “We look forward to benefiting from the harmonization products in our future clinical research activities.” “Elligo is pleased to have the opportunity to continue our work with FDA, NCATS, NCI, and data partners to apply learnings from the first phase of this project,” said Rebecca Kush, PhD, Elligo chief scientific officer. “An efficient learning health system to benefit patients will depend on broad adoption of harmonized data models and terminologies, while concurrently protecting data and reducing legal barriers to sharing research data.”
“Elligo is proud to support the FDA in this important project,” said John Potthoff, PhD, Elligo CEO. “Elligo’s mission, to enable community physicians and their patients to participate in research, will be facilitated through this work.”

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FDA approves Roche’s Rituxan (rituximab) in children with two rare blood vessel disorders

Posted by fidest press agency su giovedì, 3 ottobre 2019

Basel Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the U.S. Food and Drug Administration (FDA) has approved Rituxan® (rituximab), in combination with glucocorticoids, for the treatment of granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in paediatric patients 2 years of age and older. GPA and MPA are rare, potentially life-threatening diseases affecting small and medium sized blood vessels.
The approval is based on data from the PePRS study, a Phase IIa, global, open-label, multicentre single-arm study investigating the safety, pharmacokinetics, exploratory efficacy and pharmacodynamic outcomes of intravenous MabThera/Rituxan in 25 patients with active GPA or MPA between 6 and 17 years of age. Treatment with four weekly infusions of MabThera/Rituxan in combination with a tapering course of oral glucocorticoids was assessed in newly diagnosed or relapsing active GPA or MPA paediatric patients. Of the 25 patients in the study, 19 had GPA and 6 had MPA at baseline. Efficacy was an exploratory endpoint and primarily assessed using the Paediatric Vasculitis Activity Score (PVAS). Efficacy assessment showed that 56% of patients achieved PVAS remission by month 6, 92% by month 12, and 100% of patients achieved remission by month 18. The safety profile of MabThera/Rituxan in patients with paediatric GPA and MPA was consistent in type, nature and severity with the known safety profile of MabThera/Rituxan in adult patients with GPA and MPA, rheumatoid arthritis and pemphigus vulgaris.The FDA previously granted Priority Review to Rituxan for the treatment of GPA and MPA in paediatric patients. In 2011, Rituxan became the first and only therapy approved by the FDA for the treatment of adults with these two rare forms of vasculitis. MabThera/Rituxan is currently indicated for the treatment of four autoimmune conditions and since 2006 more than 900,000 people have been treated with MabThera/Rituxan for autoimmune conditions worldwide. MabThera/Rituxan is not indicated in children less than 2 years of age with GPA or MPA, or in children with conditions outside of GPA and MPA.

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First FDA-approved treatment designed to target both ROS1 and NTRK that also shows response in cancer that has spread to the brain

Posted by fidest press agency su martedì, 20 agosto 2019

Basel. Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the US Food and Drug Administration (FDA) has approved Rozlytrek™ (entrectinib) for the treatment of adults with ROS1-positive, metastatic non-small cell lung cancer (NSCLC). The FDA has also granted accelerated approval to Rozlytrek for the treatment of adult and paediatric patients 12 years of age and older with solid tumours that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy. These approvals are based on results from the integrated analysis of the pivotal Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA-372-001 trials, and data from the Phase I/II STARTRK-NG study. In the integrated analysis, Rozlytrek was studied in several solid tumour types, including breast, cholangiocarcinoma, colorectal, gynaecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers. In ROS1-positive, metastatic NSCLC, Rozlytrek shrank tumours in 78% of people with the disease (overall response rate [ORR]; N=51) and the duration of response (DoR) ranged from 1.8 to 36.8+ months (N=40 out of 51).1 Rozlytrek also shrank tumours in more than half of people with NTRK gene fusion-positive, locally advanced or metastatic solid tumours (ORR=57%; N=54), and objective responses were observed across 10 tumour types (DoR ranged from 2.8 to 26.0+ months; N=31 out of 54).1 Objective responses to Rozlytrek were seen in people with central nervous system (CNS) metastases at baseline.
The most common adverse reactions (≥20 percent) with Rozlytrek were fatigue, constipation, altered sense of taste (dysgeusia), swelling (oedema), dizziness, diarrhoea, nausea, nervous system disorders (dysaesthesia), shortness of breath (dyspnoea), muscle pain (myalgia), cognitive impairment, increased weight, cough, vomiting, fever (pyrexia), joint pain (arthralgia) and vision disorders. The FDA’s Accelerated Approval Program allows conditional approval of a medicine that fills an unmet medical need for a serious or life-threatening disease or condition.2 The accelerated approval for NTRK gene fusion-positive solid tumors is based on tumour response rate and durability of response, and continued approval may be contingent upon verification and description of clinical benefit in the confirmatory trials.Biomarker testing for ROS1 in NSCLC and NTRK gene fusions across all solid tumours is the only way to identify people who are eligible for treatment with Rozlytrek. Roche is leveraging its expertise in developing personalised medicines and advanced diagnostics, in conjunction with Foundation Medicine, to help identify people with ROS1 and NTRK gene fusions. Foundation Medicine will submit FoundationOne®CDx to the FDA for approval as a companion diagnostic for Rozlytrek. An FDA-approved companion diagnostic for Rozlytrek is not available at this time.

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UNITE HERE Cautions FDA Over Chinese Pharmaceutical Giant’s Purchase of Stake in GNC

Posted by fidest press agency su sabato, 17 novembre 2018

UNITE HERE has sent a letter to FDA Commissioner Dr. Scott Gottlieb, urging him to investigate the pending acquisition of a significant stake in vitamin store chain GNC by China’s Harbin Pharmaceutical Group. The letter details Harbin’s history of regulatory violations in China.The proposed transaction between Harbin and GNC was announced in February of this year. According to GNC, Harbin will invest $300 million in GNC and create a joint venture for manufacturing, marketing, sale and distribution of GNC-branded products in China. The deal would make Harbin the largest shareholder in GNC.“In China, Harbin has repeatedly been cited or sanctioned by government agencies for problems with its products – no fewer than 6 times since 2015, and three times in 2018 alone,” said Jeff Nelson, Deputy Director of Research at UNITE HERE. “Is it appropriate for a company like this to become the largest shareholder in GNC, which claims to set the standard in the nutritional supplement industry?”In January, a Chinese provincial regulator sanctioned Harbin Pharmaceutical due to the presence of ammonium in certain of its drugs in excess of regulatory limits.Reportedly, this was the second time within six months that Harbin Pharmaceutical and its subsidiaries were sanctioned for this problem.Harbin products which have been cited by Chinese regulators have included gingko pills, Siberian ginseng, and Chinese licorice. According to GNC’s website, the company sells gingko pills, Siberian root, and licorice root.“With this track record, we do not think it is appropriate for Harbin to own a substantial portion of a significant vitamin and supplement retailer in the United States. We urge the FDA to investigate this acquisition,” said Jeff Nelson.A major shareholder of Harbin, CITIC Capital, is part of the larger CITIC Group. CITIC owns one Marriott hotel in China. Viem Kwok, chairman of CITIC Resources Holdings, is the chairman of three companies which own three Marriott hotels in China, including two in Tibet.UNITE HERE represents 270,000 members working in the hotel, gaming, food service, manufacturing, textile, distribution, laundry, transportation, and airport industries in the U.S. and Canada including 17,000 employed by Marriott International. MarriottChinaObserver.org is maintained by the UNITE HERE Research Department.

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Chugai’s HEMLIBRA® Receives Regulatory Approval from U.S. FDA for Hemophilia A Without Inhibitors

Posted by fidest press agency su domenica, 7 ottobre 2018

Chugai Pharmaceutical Co., Ltd. (TOKYO:4519) announced today that the U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (US generic name: emicizumab-kxwh), a treatment for hemophilia A created by Chugai, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors, administered once weekly, every two weeks, or every four weeks. The FDA has also approved additional dosing options of every two weeks or every four weeks in adults and children with hemophilia A with factor VIII inhibitors. The US application was submitted by Genentech, a member of Roche Group.“We are very pleased that HEMLIBRA has obtained its first regulatory approval for people with hemophilia A without inhibitors,” said Chugai’s President & CEO, Tatsuro Kosaka. “Now people with hemophilia A in the US can be offered flexibility in HEMLIBRA’s dosing interval from multiple options depending on their needs, regardless of their inhibitor expression. We anticipate that HEMLIBRA will make an even greater contribution to the advancement of treatment of hemophilia A.” This regulatory approval is based on results from two Phase lll studies HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160), conducted jointly with Roche and Genentech. HAVEN 3 study was conducted to evaluate the reduction of bleed rate of HEMLIBRA subcutaneous injection once a week and once every two weeks in people with hemophilia A (12 years of age or older) without inhibitors to factor VIII. HAVEN 4 study was conducted to evaluate efficacy, safety, and pharmacokinetics of HEMLIBRA subcutaneous injection every four weeks in people with hemophilia A (12 years of age or older), with and without inhibitors to factor VIII.HEMLIBRA was granted Priority Review and Breakthrough Therapy Designation by the FDA in hemophilia A without inhibitors, following the prior designations in hemophilia A with inhibitors. Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment, prevention or diagnosis of a serious disease. In Japan and the EU, applications have been filed to regulatory authorities and are currently under review for an additional indication of prophylactic treatment for people with hemophilia A without inhibitors, as well as for additional dosage and administration as a biweekly or every four-week treatment for people with hemophilia A with inhibitors to factor VIII.

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Celltrion Completes Resubmission for Biosimilar Candidate to FDA for Review

Posted by fidest press agency su giovedì, 31 Maggio 2018

Celltrion (KRX:068270) has made a resubmission to the FDA (U.S. Food and Drug Administration) to obtain its marketing approval for CT-P10 (rituximab), a proposed mAb biosimilar to Rituxan®.
Additionally, Celltrion plans on making a resubmission for the approval of CT-P6, a proposed biosimilar to Herceptin® in June. In accordance with FDA regulations, the approval procedure will be usually finalized within six months from the resubmission, so Celltrion expects the approval for the U.S. market of the two proposed biosimilars within this year.Celltrion submitted Biologics License Applications (BLAs) for CT-P10 and CT-P6 to the U.S. FDA in April and May of last year, respectively. However, it received CRLs (complete response letter) from the FDA related to the Warning Letter issued by the FDA in January 2018, regarding the manufacturing facility in Incheon, South Korea.Celltrion has made progress addressing the concerns raised by the FDA in the Warning Letter and is committed to working with the Agency to fully resolve all outstanding issues with the highest priority and urgency.FDA has recently notified Celltrion of its re-inspection schedule regarding regular audit results, and separately from this procedure, it has confirmed the resumption of the review procedure for the two proposed biosimilars upon the resubmission of BLAs.Celltrion official said: “Celltrion is confident that CT-P10 and CT-P6, following the success of REMSIMA® (INFLECTRA®) around the globe, will be the alternative oncology treatment options for the patients in the U.S. with proven record of quality, efficacy and safety to the reference product at an affordable price.” Celltrion and Teva Pharmaceutical Industries, Ltd. entered into an exclusive partnership to commercialize CT-P10 and CT-P6 in the U.S. and Canada in October 2016. As part of the agreement, Teva is responsible for all commercial activities in the U.S. and Canada, pending regulatory approvals for both products. Celltrion has responsibility for completing all clinical development and regulatory activities.

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FDA Lifts Clinical Hold on Fitusiran

Posted by fidest press agency su sabato, 16 dicembre 2017

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today that the U.S. Food and Drug Administration (FDA) has lifted the hold on clinical studies with fitusiran, including the Phase 2 open-label extension (OLE) study and the ATLAS Phase 3 program. Alnylam and the FDA had previously reached alignment on new clinical risk mitigation measures, including protocol-specified guidelines and additional investigator and patient education concerning reduced doses of replacement factor or bypassing agent to treat any breakthrough bleeds in fitusiran studies. The FDA has now approved the protocol amendments and other updated clinical materials for fitusiran studies. Fitusiran is an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of patients with hemophilia A and B. It is designed to lower levels of AT with the goal of promoting sufficient thrombin generation to restore hemostasis and prevent bleeding.“We are pleased with the FDA’s decision to lift the clinical hold, as fitusiran holds the potential to help improve the lives of people living with hemophilia,” said Akin Akinc, Ph.D., Vice President and General Manager, Fitusiran at Alnylam. “With the additional risk mitigation measures in place, we look forward to the continued late-stage development of fitusiran and expect to resume dosing around year-end.”

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L’FDA ha approvato emicizumab di Roche per il trattamento dell’emofilia A con inibitori

Posted by fidest press agency su mercoledì, 22 novembre 2017

rocheRoche (SIX: RO, ROG; OTCQX: RHHBY) ha annunciato che la Food and Drug Administration (FDA) statunitense ha approvato il medicinale emicizumab per l’uso in profilassi, allo scopo di prevenire o ridurre la frequenza degli episodi di sanguinamento negli adulti e nei bambini affetti da emofilia A con inibitori del fattore VIII. Quasi un soggetto su tre con emofilia A severa può sviluppare inibitori a seguito del trattamento con le terapie sostitutive del fattore VIII. Questa complicanza incrementa il rischio di sanguinamenti potenzialmente letali o sanguinamenti ripetuti in grado di causare danno articolare a lungo termine. In due dei più ampi studi clinici registrativi condotti su pazienti affetti da emofilia A con inibitori, emicizumab ha dimostrato di ridurre sostanzialmente i sanguinamenti negli adulti e nei bambini.“Le persone affette da emofilia A che sviluppano inibitori affrontano notevoli difficoltà nella prevenzione dei sanguinamenti e in genere necessitano di infusioni multiple ogni settimana; e per i bambini più piccoli e le rispettive famiglie ciò può rivelarsi particolarmente gravoso”, ha dichiarato Guy Young, MD, Direttore dell’Hemostasis and Thrombosis Program presso il Children’s Hospital Los Angeles e Professore di pediatria presso la University of Southern California Keck School of Medicine, Los Angeles, California. “Questo nuovo medicinale ha dimostrato di ridurre la frequenza dei sanguinamenti rispetto ai farmaci attualmente disponibili e deve essere iniettato solo una volta a settimana. Si tratta di un trattamento che potrebbe fare la differenza per questi bambini”.
Nello studio di fase III HAVEN 1, i soggetti di età uguale o superiore a 12 anni affetti da emofilia A con inibitori, in regime di profilassi con emicizumab, hanno registrato una riduzione statisticamente significativa dei sanguinamenti trattati pari all’87% (IC al 95%: 72,3; 94,3, p < 0,0001) rispetto a quelli non sottoposti ad alcuna profilassi. In un’analisi intra-paziente unica nel suo genere, la profilassi con emicizumab ha evidenziato una riduzione statisticamente significativa dei sanguinamenti trattati pari al 79% (IC al 95%: 51,4; 91,1, p = 0,0003) rispetto al precedente trattamento con agenti bypassanti (BPA) in profilassi, nell’ambito di uno studio non interventistico (NIS) realizzato prima dell’arruolamento. Dai risultati ad interim dello studio registrativo HAVEN 2 su bambini di età inferiore a 12 anni affetti da emofilia A con inibitori è emerso che l’87% (IC al 95%: 66,4; 97,2) dei bambini sottoposti a profilassi con emicizumab ha manifestato zero sanguinamenti trattati. In un’analisi intra-paziente su 13 bambini che avevano partecipato al NIS, la profilassi con emicizumab ha evidenziato una riduzione dei sanguinamenti trattati pari al 99% rispetto al precedente trattamento con un BPA in profilassi (n = 12) o on-demand (al bisogno; n = 1). Gli eventi avversi (AE) più comuni che si sono verificati in almeno il 10% dei soggetti trattati con emicizumab sono stati reazioni in corrispondenza della sede di iniezione, mal di testa e dolore articolare (artralgia).“L’approvazione di emicizumab rappresenta un importante passo avanti per le persone affette da emofilia A con inibitori, che da tempo cercano di gestire questo disturbo emorragico e hanno dovuto attendere quasi 20 anni per poter disporre di un nuovo trattamento”, ha dichiarato Sandra Horning, MD, Chief Medical Officer e Head of Global Product Development di Roche. “Riteniamo che emicizumab potrà migliorare la protezione contro i sanguinamenti e ridurre l’impatto della somministrazione della terapia per le persone con emofilia A ed inibitori.”L’FDA ha valutato emicizumab tramite la procedura regolatoria di Priority Review (Revisione Prioritaria) e nel settembre del 2015 aveva accordato al medicinale la designazione di terapia breakthrough, nei pazienti di età uguale o superiore a 12 anni affetti da emofilia A con inibitori. L’Agenzia europea dei medicinali (EMA) sta valutando, con procedura regolatoria accelerata, i dati degli studi HAVEN 1 e HAVEN 2 ed è attualmente in corso la sottomissione ad altre autorità sanitarie di tutto il mondo.Emicizumab è ad oggi ancora oggetto di studio nell’ambito di un solido programma di sviluppo clinico che comprende altri due studi di fase III. HAVEN 3 sta valutando la profilassi con emicizumab somministrato una volta a settimana o a settimane alterne a soggetti di età uguale o superiore a 12 anni affetti da emofilia A senza inibitori del fattore VIII. HAVEN 4 sta valutando la profilassi con emicizumab somministrato ogni quattro settimane a soggetti di età uguale o superiore a 12 anni affetti da emofilia A con o senza inibitori.

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Medacta Receives FDA Clearance for Anatomic and Reverse Shoulder Systems

Posted by fidest press agency su sabato, 28 ottobre 2017

chicagoChicago. Medacta International, developer of innovative surgical techniques and products for the hip, knee, spine, and sports medicine disciplines, recently expanded its expertise to the world of shoulder surgery. The family-owned orthopedics leader today announced it has received FDA clearance for the Anatomic Shoulder and Reverse Shoulder components of its modular Medacta Shoulder System. The company also announced the successful completion of the first surgery in the United States utilizing the system, which was performed by Matthew D. Saltzman, M.D., Associate Professor of Orthopaedic Surgery at the Northwestern Memorial Hospital in Chicago, Illinois.“The procedure went very well,” said Dr. Saltzman, who specializes in shoulder and elbow surgeries and has been practicing orthopedics for 14 years. “The Medacta Shoulder System’s instrumentation and implants allow for impressive anatomic restoration, resulting in an efficient surgery and, hopefully, improved patient outcomes.”The Medacta Shoulder System, unveiled in February 2017 following its first ever surgery in Europe, is a modular solution that features a broad range of options, including wide-ranging sizes, adjustable offset, and innovative configurations. Developed by an international team of expert surgeons, the platform offers the modularity and compatibility demanded by today’s marketplace, while still respecting anatomic conversion from primary to reverse.The Shoulder System is also supported by the Medacta Orthopaedic Research and Education (M.O.R.E.) Institute’s Clinical Excellence Program and is part of a multi-center, post-marketing, prospective, open clinical study that will collect clinical and radiological outcomes for the next ten years. The M.O.R.E. Institute provides continuous educational and developmental support to Medacta surgeons as they work towards discovering new technologies and supporting patients.“The new Medacta Shoulder System is a continuation of our effort to improve orthopedic implant design and configurability for the benefit of both surgeons and their patients,” said Francesco Siccardi, Executive Vice President of Medacta International. “This latest surgery in the United States marks a milestone for Medacta in line with our company’s patient-centric vision and the same ambition that has helped us become a leader in knee, hip, and spine technology.”Medacta will launch the Shoulder System for the U.S. market at the American Academy of Orthopaedic Surgeons Annual Meeting, which will be held in New Orleans in March 2018.

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La FDA degli Stati Uniti approva benznidazole di Chemo Group

Posted by fidest press agency su martedì, 5 settembre 2017

WASHINGTON./PRNewswire/ Riguarda il trattamento dei bambini affetti dalla malattia di Chages. la Food and Drug Administration (FDA) degli Stati Uniti ha accettato la Richiesta di autorizzazione di un nuovo farmaco (NDA) presentata da Chemo Research per benznidazole. Si tratta del primo farmaco che l’FDA abbia mai approvato per il trattamento della malattia di Chagas.
Il benznidazole è un farmaco essenziale per la malattia di Chagas, una pericolosa parassitosi che secondo le stimewashington colpisce da 6 a 8 milioni di persone nel mondo. Negli Stati Uniti, si stima che in circa 300.000 convivano con la malattia di Chagas. Precedentemente il farmaco era disponibile attraverso i Centri per il Controllo e la Prevenzione delle Malattie, ma non era stato approvato dall’FDA.
Con l’approvazione del benznidazole, l’FDA ha concesso a Chemo Research un buono per il processo di revisione prioritaria (PRV) per malattie tropicali trascurate. Secondo i termini della collaborazione tra Chemo, Mundo Sano e DNDi, una parte sostanziale dei profitti derivati dalla futura vendita del PRV sarà investita nel potenziamento dell’accesso al trattamento da parte dei pazienti affetti dalla malattia di Chagas e al miglioramento della salute dei pazienti di altre aree patologiche.”Pochissime persone affette dalla malattia di Chagas hanno acceso alle cure a livello globale, e negli Stati Uniti si contano sulle dita delle mani”, racconta Bernard Pécoul, Executive Director di DNDi. “Speriamo che la registrazione presso l’FDA spingerà anche i Paesi endemici dell’America Latina a intraprendere lo stesso percorso. In definitiva, crediamo che questo contribuirà a trasformare la dinamica dell’accesso alle cure in tutte le Americhe.”
Guardando al futuro, Chemo Group continuerà a lavorare in collaborazione con Exeltis, Mundo Sano e Drugs for Neglected Diseases initiative (DNDi) per superare gli ostacoli nel trattamento della malattia di Chagas. Exeltis sarà responsabile della distribuzione del prodotto sul mercato statunitense. Parallelamente, Mundo Sano e DNDi profonderanno ogni sforzo per potenziare l’accesso alle cure e sensibilizzare i pazienti.

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FDA Grants Priority Review to Genentech’s Emicizumab for Hemophilia A with Inhibitors

Posted by fidest press agency su lunedì, 28 agosto 2017

emofilia-geneticaGenentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) and granted Priority Review for emicizumab prophylaxis (preventative) as a once-weekly subcutaneous treatment for adults, adolescents and children with hemophilia A with factor VIII inhibitors. Nearly one in three people with hemophilia A develop inhibitors to standard factor VIII replacement therapies, which limits treatment options and increases the risk of life-threatening bleeds and repeated bleeds, particularly in joints, that cause long-term damage. “Genentech has a history of developing innovative antibody therapies to address some of the highest unmet medical needs,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “Results of our Phase III study in adults and adolescents as well as early Phase III results in children showed that emicizumab has significant potential to help people with hemophilia A with inhibitors, who face major challenges in preventing and treating bleeds. We are working with the FDA to hopefully bring this new prophylactic treatment option to the hemophilia A inhibitor community as soon as possible.” The BLA for emicizumab is based on results from the Phase III HAVEN 1 study in adults and adolescents 12 years of age and older, as well as interim results from the Phase III HAVEN 2 study in children younger than 12 years of age. Results from HAVEN 1 were published in The New England Journal of Medicine (NEJM) and results from both studies were presented at the 26th International Society on Thrombosis and Haemostasis (ISTH) Congress in July 2017.The FDA is expected to make a decision on approval by February 23, 2018. Priority Review designation is granted to medicines that the FDA has determined to have the potential to provide significant improvements in the safety and effectiveness of the treatment, prevention or diagnosis of a serious disease. The FDA granted Breakthrough Therapy Designation for emicizumab in adults and adolescents with hemophilia A with inhibitors in September 2015. Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate substantial improvement over existing therapies.
Data from both HAVEN 1 and HAVEN 2 have also been submitted for approval consideration to the European Medicines Agency (EMA) and will be reviewed under accelerated assessment. Additional studies evaluating emicizumab in people with hemophilia A both with and without inhibitors and exploring less frequent dosing regimens are ongoing.

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Oppioidi e benzodiazepine, i rischi di un uso combinato denunciati dalla Fda

Posted by fidest press agency su martedì, 13 settembre 2016

droga cannabis-vicinoSono gravi i rischi di un uso combinato di oppioidi e benzodiazepine: lo ha sottolineato la Food and drug administration (Fda), che in una comunicazione di sicurezza ha chiesto di rafforzare le avvertenze contenute nell’etichettatura di questi farmaci. La decisione di informare medici e pazienti è giunta dopo un’ampia revisione delle ultime evidenze scientifiche che hanno riportato effetti avversi di varia natura: sonnolenza estrema, depressione respiratoria, coma e decesso. I cambiamenti richiesti riguardano circa 400 prodotti e sono parte del piano d’azione che l’Agenzia regolatoria americana ha avviato sul tema degli oppioidi, con l’intento di stabilire e diffondere le regole per un utilizzo sicuro e di contrastare l’abuso di questi farmaci, fenomeno già più volte segnalato negli Stati Uniti.La revisione ha infatti documentato una crescita delle prescrizioni per lo stesso paziente di farmaci appartenenti alle due classi citate e anche un numero sempre maggiore di eventi avversi. Dal 2004 al 2011 sono aumentate le visite al pronto soccorso dovute all’utilizzo di oppioidi e benzodiazepine e per entrambe le classi di farmaci si sono avuti decessi per overdose, che sono addirittura triplicati nel periodo preso in esame. Anche l’uso contemporaneo di questi farmaci è in forte crescita e oggi riguarda oltre due milioni e mezzo di cittadini statunitensi, con un aumento di oltre il 40% tra il 2002 e il 2014.
«È a dir poco un problema di salute pubblica; – ha detto il Commissario della Fda Robert Califf – assistiamo a un aumento sostanziale di sovradosaggi e di decessi evitabili. Imploriamo gli operatori sanitari di ascoltare questi nuovi avvertimenti e di valutare con più attenzione, attraverso un’analisi specifica per ogni paziente, se i benefici di un uso combinato di oppioidi, benzodiazepine, o qualunque altro farmaco depressivo del Snc, siano superiori rispetto ai gravi possibili rischi». Se negli Usa gli effetti avversi gravi sono segnalati in crescita, dal punto di vista clinico è noto da tempo che l’uso combinato di oppioidi e benzodiazepine, entrambi depressori del sistema nervoso centrale, è rischioso. E nell’Unione europea, le informazioni su questi prodotti sono già presenti. (fonte: farmacista33) (foto: pillole)

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La Food and Drug Administration (FDA) statunitense ha approvato idarucizumab

Posted by fidest press agency su mercoledì, 21 ottobre 2015

Boehringer Ingelheim, Germania. Boehringer Ingelheim GmbH comunica che la Food and Drug Administration (FDA) statunitense ha approvato idarucizumab, per l’uso in pazienti trattati con dabigatran etexilato, che necessitano di un’inattivazione dell’effetto anticoagulante, in caso di interventi chirurgici d’emergenza o procedure d’urgenza, oppure in caso di sanguinamento non controllato che possa mettere a rischio la vita del paziente.
L’FDA ha concesso a idarucizumab la designazione di Breakthrough Therapy con una “Priority Review”. Il farmaco è stato approvato con procedura accelerata. Il dossier della richiesta di autorizzazione ha incluso i risultati di studi su volontari sani e i risultati dell’analisi intermedia dello studio clinico RE-VERSE AD™ (NCT 02104947). In questi studi, l’inattivazione dell’effetto anticoagulante di dabigatran realizzata da idarucizumab si è vista immediatamente, a pochi minuti dalla somministrazione di 5 grammi del farmaco. Inoltre, non è stato osservato alcun effetto pro-coagulante dopo la somministrazione di idarucizumab.“L’arrivo dei nuovi anticoagulanti orali (NAO) ha segnato un progresso importante nella terapia anticoagulante. Benché esistano strategie generali per la gestione di sanguinamenti in caso di terapia con essi, sino ad oggi non era disponibile alcun farmaco che inattivasse in maniera immediata e specifica il loro effetto anticoagulante, in quelle rare situazioni di emergenza in cui la rapidità d’azione è importante, quali ad esempio un sanguinamento che mette a rischio la vita del paziente o la necessità di procedere rapidamente a un’operazione chirurgica o altro intervento” ha dichiarato il Dottor Charles Pollack, Professore di Medicina d’Emergenza-Urgenza, Sidney Kimmel Medical College, Università Thomas Jefferson di Philadelphia, USA, e Principale Sperimentatore dello studio RE-VERSE AD™. “L’avere ora a disposizione idarucizumab significa disporre di una soluzione unica per inattivare l’azione anticoagulante in pazienti che assumono dabigatran”.
Boehringer Ingelheim è impegnata per rendere disponibile idarucizumab quanto più ampiamente possibile. Il farmaco è attualmente in fase d’esame da parte di diverse autorità regolatorie, compresa la canadese Health Canada. Sono in corso ulteriori richieste di autorizzazione del farmaco alle autorità regolatorie di altri paesi.
Idarucizumab è un frammento di anticorpo umanizzato, o Fab, sviluppato come farmaco specifico per inattivare l’effetto di dabigatran. Idarucizumab si lega in maniera specifica esclusivamente alle molecole di dabigatran, neutralizzandone l’effetto anticoagulante senza interferire con la cascata della coagulazione. Idarucizumab è approvato negli Stati Uniti dove è indicato per l’impiego in pazienti trattati con dabigatran, che necessitano un’inattivazione dell’effetto anticoagulante dello stesso in caso di:
• interventi chirurgici d’emergenza/procedure urgenti
• sanguinamento non controllato o che possa mettere a rischio la vita del paziente
In altri paesi idarucizumab è attualmente in fase d’esame da parte delle autorità regolatorie o sono in corso gli inoltri delle richieste di registrazione. Idarucizumab è l’unico farmaco che svolge un’azione di inattivazione specifica dell’effetto di un NAO a essere in fase d’esame da parte delle autorità regolatorie. Boehringer Ingelheim intende inoltrare le richieste di autorizzazione all’immissione in commercio per idarucizumab in tutti i paesi in cui dabigatran è approvato.

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Fda approva pillola dei “5 giorni dopo”

Posted by fidest press agency su lunedì, 30 agosto 2010

La Food and drug administration (Fda) ha approvato ulipristal acetato (Ella), farmaco per la contraccezione d’emergenza dei “5 giorni dopo”. l’Ente ha specificato che l’approvazione è esclusiva per l’impiego dietro presentazione di ricetta medica e che il farmaco essere assunto entro 120 ore dopo un rapporto sessuale non protetto o dopo il fallimento di un altro metodo anticoncezionale. Non deve essere considerato un sistema contraccettivo di routine. Il farmaco è prodotto da Laboratoire Hra Pharma e distribuito negli Stati Uniti da Watson Pharma ed è disponibile in Europa dal maggio 2009 sotto il nome di EllaOne, «la sua sicurezza ed efficacia» spiega una nota Fda «sono state dimostrate in due trial clinici di fase III. Gli eventi avversi più frequenti durante gli studi sono stati mal di testa, nausea, dolore addominale, dolore durante il ciclo, senso di affaticamento». (fonte farmacista33)

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