Ipsen
Biopharmaceutical Manufacturer
Argenx
Antibody Drug Developer
Sanofi
Pharmaceutical Manufacturer
Advances in Rare Disease Drug Development
☞ Ipsen's New Drug for Primary Biliary Cholangitis Approved for Marketing in the United States
☞ Roche's PNH New Drug Approved for Marketing in the United States
☞ Sarepta's DMD Gene Therapy Receives FDA Approval for Expanded Indications
☞ Argenx's Efgartigimod Approved for New Indication in the United States
☞ Sanofi's Hemophilia A Therapy Approved for Marketing in the EU
☞ Roche's Multiple Sclerosis Antibody Therapy Approved for Marketing in the EU
☞ Pfizer's Hemophilia Gene Therapy Receives Conditional Marketing Authorization from EMA
01
Ipsen's New Drug for Primary Biliary Cholangitis Approved for Marketing in the United States
Ipsen Announces FDA Approval of Iqirvo (Elafibranor) for the Treatment of Primary Biliary Cholangitis (PBC) in Adults with Inadequate Response to or Intolerance of UDCAIpsen announced that its product Iqirvo (Elafibranor) has received FDA approval for use in combination with ursodeoxycholic acid (UDCA) to treat adults with primary biliary cholangitis (PBC) who have an inadequate response to UDCA or are intolerant to UDCA as monotherapy. Elafibranor is a once-daily, dual peroxisome proliferator-activated receptor (PPAR) α/δ agonist that helps reduce bile acid production and alleviates inflammation and fibrosis by targeting and activating PPARα/δ.
02
Roche's PNH New Drug Approved for Marketing in the United States
Roche announced that its targeted C5 complement protein Piasky (Crovalimab) has been approved by the FDA for marketing to treat patients with paroxysmal nocturnal hemoglobinuria (PNH). Crovalimab is a humanized monoclonal antibody administered once monthly via subcutaneous injection. By binding to C5, it induces the degradation of the C5 protein, thereby blocking the complement pathway and exerting therapeutic effects.
03
Sarepta's DMD Gene Therapy Receives FDA Approval for Expanded Indications
Sarepta Therapeutics announced that its gene therapy Elevidys (delandistrogene moxeparvovec, SRP-9001), co-developed with Roche, has received FDA approval for an expanded indication to treat Duchenne muscular dystrophy (DMD) patients aged 4 years and older who can walk independently. It also received accelerated approval for DMD patients aged 4 years and older who cannot walk independently. Previously, the therapy was only approved for use in children aged 4-5 years with DMD who could walk independently. Elevidys delivers a transgene encoding micro-dystrophin packaged in an AAV vector, administered via a single intravenous injection, enabling muscle production of a recombinant protein with partial dystrophin functionality.
04
Argenx Efgartigimod Approved for New Indication in the United States
Argenx announced that its developed subcutaneous injection of Efgartigimod (VYVGART Hytrulo) has received FDA approval for the treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Efgartigimod is a once-weekly FcRn antagonist that can reduce patients' antibody levels by inhibiting IgG reabsorption, decrease various antibodies in circulation, thereby exerting therapeutic effects on autoimmune-mediated diseases.
05
Sobi Announces EC Approval of Altuvoct (Efanesoctocog Alfa) for Routine Prophylaxis, On-Demand Treatment to Control Bleeding Episodes, and Perioperative Management in Adults and Children with Hemophilia ASobi announced that Altuvoct (Efanesoctocog alfa), jointly developed with Sanofi, has received approval from the European Commission for marketing. It is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes in adults and children with Hemophilia A, as well as perioperative management (surgical procedures). Efanesoctocog alfa is a high-persistence Factor VIII replacement therapy based on Fc fusion technology, incorporating von Willebrand factor and a polypeptide, significantly extending the drug’s duration in circulation.
06
Roche's Multiple Sclerosis Antibody Therapy Approved for Marketing in the EU
Roche announced that its targeted CD20 antibody therapy Ocrevus (ocrelizumab) has been approved by the European Commission for the treatment of relapsing multiple sclerosis (RMS) and primary progressive multiple sclerosis (PPMS). Ocrevus is a humanized monoclonal antibody that requires only two subcutaneous injections per year to completely suppress clinical relapses and brain lesions in multiple sclerosis patients. It works by binding to the CD20 protein on the surface of B cells, thereby maintaining essential functions of the patient's immune system.
07
Pfizer's Hemophilia Gene Therapy Receives Conditional Marketing Authorization from EMA
Pfizer announced that its one-time gene therapy Durveqtix (fidanacogene elaparvovec) has received conditional marketing authorization from the EMA for the treatment of adult patients with moderate to severe and severe Hemophilia B who do not have Factor IX (FIX) inhibitors in their bodies and have not detected antibodies against the adeno-associated virus serotype Rh74 variant. Durveqtix is a novel gene therapy composed of a bioengineered adeno-associated virus capsid and a transgene encoding a highly active FIX variant, which enables Hemophilia B patients to produce autologous FIX protein after administration, thereby achieving therapeutic effects. This therapy was approved for marketing in the United States in April this year.
Rare Neurological Disorders Communication Group
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