Biotek-snakken — Januar 2021
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Orphazyme disclosed that the FDA has confirmed that the NDA remains under Priority Review. The FDA grants Priority Review to applications for potential therapies that, if approved, could offer a significant improvement in safety or effectiveness, diagnosis, or prevention of serious conditions. Notably, the FDA has confirmed that the extension does not impede eligibility for a Pediatric Rare Disease Priority Review Voucher.
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Arimoclomol previously received the FDA's Fast-Track and Breakthrough Therapy Designations for NPC as well as Orphan Drug and Rare Pediatric Disease Designations. If approved in the U.S., arimoclomol will be the first and only approved medicine for NPC. In November 2020, Orphazyme also submitted a Marketing Authorisation Application to the European Medicines Agency for arimoclomol to treat NPC.
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Orphazyme's U.S. President issued the following statement on December 27, 2020:
"Orphazyme is working closely with the FDA to support the final review of the new drug application for arimoclomol. There is significant unmet medical need for the treatment of NPC, and we are committed to bringing arimoclomol to patients in the U.S. and Europe as soon as possible."
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Orphazyme's Chief Medical Officer added:
"We have responded to all FDA information requests and submitted all outstanding information regarding the arimoclomol NDA for NPC. The Phase 3 trials for Amyotrophic Lateral Sclerosis and Inclusion Body Myositis remain on track for read-out in the first half of 2021 and we look forward to providing an update on our progress."
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Steven A. Kriegsman, Chairman and Chief Executive Officer of CytRx, commented:
"We believe Orphazyme has taken a number of important steps in 2020 ahead of potential commercialization of arimoclomol for NPC upon prospective FDA approval during the first half of 2021. Orphazyme has strengthened its financial position through a successful capital raise and subsequently established a strong U.S. footprint with new headquarters in Chicago and the addition of more than 30 employees.
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In our view, Orphazyme is well positioned for future distribution and expanded engagement with providers, patients, regulators and the clinical testing community in 2021. It is also noteworthy that next year, Orphazyme may receive a response to its submission for regulatory approval in Europe for arimoclomol to treat NPC."
We will continue to provide updates that are relevant to our agreement with Orphazyme.
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Reklame: https://www.proinvestor.com/donation
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Tak @Fandel
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Det spørger jeg til 30/12 19:49? Bioport har åbenbart valgt at lade sig garanterer til en egengodkendelse eftersom der henvises til CE godkendelse? Den hæftelse er meget kraftigere end en egentlig godkendelse - og lovlig hvis een er korrekt med ens produkt!
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