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FDA Accepts Alkermes Resubmission of New Drug Application for ALKS 3831

FDA Accepts Alkermes Resubmission of New Drug Application for ALKS 3831 - FDA Sets PDUFA Target Action Date of June 1, 2021 - DUBLIN, Dec. 29, 2020 /PRNewswire/ Alkermes plc (Nasdaq: ALKS) today announced that the U.S. Food and Drug Administration (FDA) has acknowledged receipt of the company s New Drug Application (NDA) resubmission for ALKS 3831 (olanzapine/samidorphan) for the treatment of adults with schizophrenia and adults with bipolar I disorder, and has assigned the application a new Prescription Drug User Fee Act (PDUFA) target action date of June 1, 2021. The FDA classified the resubmission as a complete, Class 2 response to the Complete Response Letter (CRL) issued in November 2020, following a remote review of records requested under Section 704(a)(4) of the Federal Food, Drug, and Cosmetic Act (the FDCA ) relating to the manufacture of ALKS 3831 at the company s Wilmington, OH facility. Subsequent to Alkermes resubmission of the NDA, the FDA issued a new request

LORBRENA® (lorlatinib) sNDA in Previously Untreated ALK-Positive Lung Cancer Accepted for Priority Review by U S FDA

Press release content from Business Wire. The AP news staff was not involved in its creation. LORBRENA® (lorlatinib) sNDA in Previously Untreated ALK-Positive Lung Cancer Accepted for Priority Review by U.S. FDA December 28, 2020 GMT NEW YORK (BUSINESS WIRE) Dec 28, 2020 Pfizer Inc. (NYSE:PFE) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for LORBRENA ® (lorlatinib) as a first-line treatment for people with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC). The sNDA is based on data from the pivotal CROWN study and is being reviewed by the FDA under its Real-Time Oncology Review (RTOR) pilot program. The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is in April 2021.

Orphazyme provides regulatory update on arimoclomol for NPC

[December 27, 2020] ­­Orphazyme A/S Company announcement                                                                                        No. 73/2020 Company Registration No. 32266355 Copenhagen, Denmark – December 27, 2020 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, announced today the U.S. Food and Drug Administration (FDA) has extended the review period of the New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC) by a standard extension period of three months. This extension is necessary for the FDA to complete its review. The updated Prescription Drug User Fee Act (PDUFA) target action date is June 17, 2021.  The FDA has confirmed the NDA remains und

Chiesi Global Rare Diseases Announces First Patient Treated in Expanded Access Program for

Press release content from Globe Newswire. The AP news staff was not involved in its creation. Chiesi Global Rare Diseases Announces First Patient Treated in Expanded Access Program for . Chiesi USA, Inc.December 23, 2020 GMT - 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 - BOSTON, Dec. 23, 2020 (GLOBE NEWSWIRE) Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare group (Chiesi Group), 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 Nol

Amryt Granted Orphan Drug Designation by the FDA for AP103

Press release content from Globe Newswire. The AP news staff was not involved in its creation. Amryt Granted Orphan Drug Designation by the FDA for AP103 Amryt Pharma plcDecember 23, 2020 GMT Amryt Granted Orphan Drug Designation by the FDA for AP103 DUBLIN, Ireland, and Boston MA, December 23, 2020, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from serious and life-threatening rare diseases, is pleased to announce today that the U.S. Food and Drug Administration (“FDA”) has granted orphan drug designation for AP103 for the treatment of Dystrophic Epidermolysis Bullosa (“DEB”).  AP103 is based on Amryt’s gene-therapy platform technology and offers a potential treatment for patients with DEB, a subset of EB.  

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