Chiesi Global Rare Diseases Announces First Patient Treated in Expanded Access Program for Pegunigalsidase Alfa for Proposed Treatment of Fabry Disease
- Program grants access to investigational therapy for Fabry disease patients in the United States who cannot be adequately treated with currently available FDA-approved drugs -
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BOSTON, Dec. 23, 2020 /PRNewswire/ Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare group (Chiesi Group)
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today announced the first patient has been treated in the Expanded Access Program (EAP) in the United States for pegunigalsidase alfa, currently in development for the treatment of Fabry disease. Treatment was initiated on December 21, 2020 by Ozlem Goker-Alpan, M.D., founder and Chief Executive Officer of the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC) in Fairfax, Va, and Lauren Noll, Clinical Resea
Novartis Gets CRL From FDA Due To Unresolved Facility Inspection-related Conditions On Inclisiran
BASEL (dpa-AFX) - Novartis (NVS) said that it received a complete response letter from the U.S. Food and Drug Administration regarding the new drug application for inclisiran. It is due to unresolved facility inspection-related conditions.
The FDA did not raise any concerns related to the efficacy or safety of inclisiran, the company said in a statement.
inclisiran is a potential treatment for hyperlipidemia in adults who have elevated low-density lipoprotein cholesterol (LDL-C) while being on a maximum tolerated dose of a statin therapy.
According to Novartis, the FDA stated that the agency cannot approve the NDA by the Prescription Drug User Fee Act (PDUFA) action date of December 23, 2020, due to unresolved facility inspection-related conditions. The conditions will be conveyed to the European manufacturing facility within 10 business days.
Provided by Dow Jones By Michael Dabaie FibroGen Inc. shares were down 14% to $37.66 in late morning trading. The company said after the market close Friday that the Food and Drug Administration extended the review period of the new drug application for roxadustat for the treatment of anemia of chronic kidney disease by three months. The updated Prescription Drug User Fee Act action date is March 20, 2021, the company said. FibroGen said the FDA is close to finalizing its review of the NDA. FibroGen is submitting additional analyses of existing roxadustat clinical data, which require an extension of the original PDUFA date.
Novartis Receives FDA Complete Response Letter on Inclisiran medscape.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from medscape.com Daily Mail and Mail on Sunday newspapers.
By Stephen Northrup Dec. 21, 2020Reprints The investigation by Sen. Michael Enzi (R-Wyo.) into the approval and subsequent withdrawal of Vioxx led to sweeping changes in ensuring drug safety.
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The prescription painkiller originally known as Vioxx may be making a comeback after its maker, Merck, abruptly pulled it from the market in 2004 following studies that showed the drug roughly doubled patients’ risks of heart attack and stroke and may have contributed to an estimated 60,000 deaths.
According to STAT, Tremeau Pharmaceuticals, a privately held Massachusetts drug company, is about to begin clinical trials of rofecoxib (the generic name for Vioxx) as a treatment for hemophilic arthropathy, a persistent joint disease for which doctors often prescribed Vioxx off-label.