🎊 New Gene Therapy for Duchenne’s Approved

1/ The FDA has granted accelerated approval to Sarepta Therapeutics' gene therapy, SRP-9001 marketed as Elevidys, for boys aged 4 and 5 suffering from Duchenne muscular dystrophy (DMD).

2/ Elevidys it is priced at $3.2 million per patient, making it the second most expensive medicine in the United States.

3/ Elevidys is the first in vivo gene therapy to receive accelerated approval with limited data (EMBARK Clinical trial). This has garnered attention from rare disease advocates and drug developers, who are observing the FDA’s flexibility in granting accelerated approvals for fatal diseases based on biomarker data.

4/ Elevidys uses a viral vector to deliver a copy of a micro-dystrophin gene to muscle cells. While not a cure, the goal is to have patients experience outcomes similar to those with Becker muscular dystrophy, a milder form of muscle disease.

5/ Elevidys is not suitable for patients who have pre-existing antibodies against the viral vector used, rAAVrh74, or mutations at exons 8 and/or 9 in the dystrophin gene. This makes approximately 14% of DMD patients ineligible for the therapy.

✋🏼 Myasthenia Gravis: New Drug Approved

1/ The Dutch biotech company, Argenx, received FDA approval for an easier-to-administer version of its autoimmune disease drug efgartigimod, which will be marketed as Vyvgart Hytrulo.

2/ Vyvgart Hytrulo will be available in July and is targeted at patients with generalized myasthenia gravis, a condition where the immune system attacks muscles, causing difficulty in movement, speaking, and swallowing.

3/ Both the subcutaneous injection and intravenous infusion versions of Vyvgart are priced the same, around $225,000 per year for a typical patient.

4/ Vyvgart targets and lowers the levels of immunoglobulin G antibodies, which includes those causing autoimmune disease. This reduces muscle weakness but may increase the risk of infections.

5/ In clinical trials, 68% of patients receiving Vyvgart infusions showed significant improvement in daily activities compared to 30% who received a placebo.

🩸Novel Drug in Phase III testing for Polycythemia Vera

1/ Protagonist Therapeutics is developing a drug called rusfertide to treat polycythemia vera, a rare blood cancer characterized by the overproduction of red blood cells.

2/ At the European Hematology Association’s annual meeting, researchers presented promising Phase 2 results of rusfertide, showing that it helped stabilize red blood cell levels and improved patients’ symptoms such as fatigue and brain fog.

3/ In the Phase 2 trial, patients receiving weekly injections of rusfertide had better control of their hematocrit levels, were less likely to need phlebotomy, and reported improvements in concentration, energy, and activity levels.

4/ Protagonist Therapeutics expects to complete enrollment for its 250-person Phase 3 study by the end of the year.

🔴 ALS Drug Hits a Wall, At Least in EU

1/ The European Medicines Agency (EMA) committee issued a negative recommendation on Amylyx Pharmaceuticals' ALS (Amyotrophic Lateral Sclerosis) drug, Albrioza, indicating it may not be approved for the European market.

2/ Albrioza, also known as AMX0035 and marketed in the U.S. as Relyvrio, has been approved in the United States and Canada, and has been welcomed by many ALS patients.

3/ In a small trial, ALS patients who received Relyvrio for 24 weeks scored about two points better on a 48-point scale of symptoms compared to those on a placebo.

4/ Initially, the U.S. Food and Drug Administration (FDA) had reservations regarding the approval of the drug, and an advisory panel initially concluded that the results did not demonstrate the drug’s effectiveness. However, after advocacy from patient groups, the FDA reconvened the advisory panel, which then endorsed the drug.

5/ Amylyx has argued that its trial data merits conditional approval in Europe and has expressed its commitment to make the treatment available in Europe.