March 5, 2024

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FDA approved 5 new drugs In February:  4 of which were orphan drugs

FDA approved 5 new drugs In February:  4 of which were orphan drugs


FDA approved 5 new drugs In February:  4 of which were orphan drugs.

In February, FDA’s Center for Drug Evaluation and Research (CDER) and Center for Biologics Evaluation and Research (CBER) approved a total of 5 new drugs, of which rare disease drugs (orphan drugs) accounted for 4 of them.


FDA approved 5 new drugs In February:  4 of which were orphan drugs




In February, the FDA approved 4 drugs for rare diseases:

  • On February 16, Chiesi Global Rare Diseases’ Lamzede (velmanase alfa-tycv) was approved for the treatment of non-CNS manifestations of alpha-mannosidosis (AM) in adult and pediatric patients, becoming the first FDA-approved treatment Enzyme replacement therapy for this disease.


  • On February 17, Travere Therapeutics’ Filspari (sparsentan) was approved to reduce proteinuria in patients with IgA nephropathy at high risk of disease progression, and is the first non-immunosuppressive therapy approved by the FDA for the treatment of IgA nephropathy.


  • On February 23, the FDA approved Sanofi’s Altuviiiio (efanescoctcog alfa) for routine prophylaxis and on-demand treatment in adults and children with hemophilia A to control bleeding episodes and perioperative management.


  • On February 28, Reata Pharmaceuticals announced that the FDA approved its Skyclarys (omavaloxolone) for the treatment of Friedrich’s ataxia in adolescents aged 16 and above. Drugs for Greek ataxia patients.



The only non-rare disease new drug approved in February is GlaxoSmithKline’s hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) Jesduvroq (daprodustat, daprusstat), February 1 Received FDA approval as the first marketed oral treatment in the U.S. for the treatment of anemia caused by chronic kidney disease (CKD) in adults on dialysis for at least four months.



In addition, there are 2 new drugs that have been rejectedby the FDA:

  • On February 14, Soligenix announced that it had received a rejection (RTF) letter from the FDA regarding its new drug application (NDA) for its synthetic hypericin photodynamic therapy HyBryte (synthetic hypericin) in the treatment of early-stage cutaneous T-cell lymphoma (CTCL) . Following an initial review by the FDA, it was determined that the Company’s NDA submitted on December 14, 2022 was not complete enough for substantive review.


  • On February 28, Cytokinetics announced that it had received a New Drug Application (NDA) from the U.S. Food and Drug Administration (FDA) for omecamtiv mecarbil for the treatment of heart failure with reduced ejection fraction (HFrEF). Complete Response Letter (CRL) . The FDA said the GALACTIC-HF study on which the NDA application was based was not sufficiently convincing to demonstrate its effectiveness in reducing the risk of heart failure events and cardiovascular death in adults with HFrEF. Results from additional clinical trials of omecamtiv mecarbil are needed to establish substantial evidence of an effective treatment for HFrEF, where the benefits outweigh the risks, the FDA said.




FDA approved 5 new drugs In February:  4 of which were orphan drugs

(source:internet, reference only)

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Important Note: The information provided is for informational purposes only and should not be considered as medical advice.