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RSS FeedPosted by Michael Wonder on 25 Mar 2026
FDA approves drug to treat neurologic manifestations of Hunter syndrome
25 March 2026 - The US FDA approved Avlayah (tividenofusp alfa-eknm) to treat certain individuals with Hunter syndrome (mucopolysaccharidosis type II ...
Posted by Michael Wonder on 01 Mar 2026
US FDA approves BioMarin’s Palynziq (pegvaliase-pqpz) for adolescents 12 years of age and older with phenylketonuria
27 February 2026 - BioMarin today announced that the US FDA has approved the company's supplemental biologics license application for Palynziq ...
Posted by Michael Wonder on 24 Feb 2026
US FDA has granted accelerated approval of Loargys (pegzilarginase-nbln) for the treatment of hyperargininemia in patients 2 years and older with arginase 1 deficiency
23 February 2026 - Immedica Pharma today announced that the US FDA has granted accelerated approval of Loargys (pegzilarginase-nbln), an ...
Posted by Michael Wonder on 24 Feb 2026
FDA launches framework for accelerating development of individualised therapies for ultra-rare diseases
23 February 2026 - The U.S. FDA today issued draft guidance for sponsors seeking approval for targeted individualised therapies by ...
Posted by Michael Wonder on 01 Feb 2026
Ultragenyx resubmits biologics license application for UX111 AAV gene therapy to treat Sanfilippo syndrome type A (MPS IIIA) to US FDA
30 January 2026 - Company expects up to six month review period per FDA guidelines. ...
Posted by Michael Wonder on 03 Nov 2025
US FDA approves Kygevvi (doxecitine and doxribtimine), the first and only treatment for adults and children living with thymidine kinase 2 deficiency
3 November 2025 - UCB today announced that Kygevvi has been granted approval by the US FDA for the treatment ...
Posted by Michael Wonder on 21 Aug 2025
Rhythm Pharmaceuticals announces FDA acceptance of sNDA for setmelanotide in acquired hypothalamic obesity
20 August 2025 - US FDA accepts sNDA for filing with priority review; sets PDUFA goal date of 20 December 2025. ...
Posted by Michael Wonder on 03 Mar 2025
Supporting the development of drugs for rare diseases - the importance of regulatory transparency
1 March 2025 - Transparency regarding the information submitted to the FDA and the agency’s decision making could have far-reaching ...
Posted by Michael Wonder on 03 Mar 2025
Vertex receives CHMP positive opinion for expanded label for Kaftrio in combination with ivacaftor for people with cystic fibrosis to include rare mutations
28 February 2025 - If approved, approximately 4,000 people with cystic fibrosis in the European Union will be eligible for ...
Posted by Michael Wonder on 09 Jan 2025
Denali Therapeutics announces US FDA breakthrough therapy designation granted to tividenofusp alfa for the treatment of Hunter syndrome
8 January 2025 - Denali Therapeutics today announced that the US FDA has granted breakthrough therapy designation for tividenofusp alfa (DNL310) ...
Posted by Michael Wonder on 22 Dec 2024
Vertex announces US FDA approval of Alyftrek, a once daily next in class CFTR modulator for the treatment of cystic fibrosis
20 December 2024 - In head to head clinical trials, Alyftrek was non-inferior on ppFEV1 and further decreased sweat chloride ...
Posted by Michael Wonder on 22 Dec 2024
Vertex announces US FDA approval for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include additional non-F508del Trikafta responsive variants
20 December 2024 - Approximately 300 more people with cystic fibrosis in the US are now eligible for a medicine ...
Posted by Michael Wonder on 21 Dec 2024
Ultragenyx submits biologics license application to the US FDA for UX111 AAV gene therapy for the treatment of Sanfilippo syndrome type A
19 December 2024 - If approved, UX111 would be the first approved therapy in the US for Sanfilippo syndrome type ...
Posted by Michael Wonder on 14 Dec 2024
FDA approves new treatment for congenital adrenal hyperplasia
13 December 2024 - Today, the US FDA approved Crenessity (crinecerfont) to be used together with glucocorticoids to control androgen levels ...
Posted by Michael Wonder on 10 Dec 2024
Chiesi Global Rare Diseases and Protalix BioTherapeutics announce validation of variation submission by EMA for pegunigalsidase alfa
9 December 2024 - Application to label a less frequent dosing regimen at a dose of 2 mg/kg body weight ...