Update for September 29, 2022

The FDA approved elivaldogene autotemcel (Skysona, bluebird bio), on 9/16/2022, to slow the progression of neurologic dysfunction in early, active cerebral adrenoleukodystrophy (CALD). Elivaldogene autotemcel will only be available at Qualified Treatment Centers (QTC), selected by Bluebird. The cost for the one-time treatment is $3 million. Unlike betibeglogene autotemcel, bluebird bio will not offer an outcomes-based payment plan with rebates for patients that do not respond for elivaldogene autotemcel.
 
In September 2022, the FDA allowed Merck to proceed with two HIV-1 trials evaluating a lower dose of islatravir in combination with doravirine and a second trial in combination with lenacapavir.
 
The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 13-4 to recommend RBX2660 to reduce recurrent C. difficile infection after antibiotic treatment.
 
Vertex and CRISPR Therapeutic initiated a rolling BLA submission for exagamglogene autotemcel for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
 
The FDA has placed a hold on the Phase III VITESSE trial, evaluating Viaskin Peanut, until DBV makes changes to the study protocol regarding statistical analysis and total number of patients.
 
The FDA has designated dovitinib a Rare Pediatric Disease treatment for osteosarcoma.
 
Botanix Pharmaceuticals submitted an NDA for sofpironium bromide gel for the treatment of severe primary axillary hyperhidrosis.
 
The European Commission (EC) approved:

• Faricimab (Vabysmo, Roche) for the treatment of neovascular macular degeneration and visual impairment due to diabetic macular edema.
• Tezepelumab (Tezspire) as add-on maintenance treatment of severe asthma in patients who are inadequately controlled with high dose inhaled corticosteroids plus another medicinal product.
• Fosdenopterin (Nulibry, BridgeBio Pharma and Sentynl Therapeutics) to reduce the risk of death due to Molybdenum Cofactor Deficiency Type A.​
  

CHMP recommended approval of:

• Nirsevimab (Beyfortus, AstraZeneca & Sanofi) for the prevention of respiratory syncytial virus lower respiratory tract disease in newborns and infants.
• Sutimlimab (Enjaymo, Sanofi) for the treatment of hemolytic anaemia.
• Maribavir (Livtencity, Takeda, ViroPharma, Shire) for the treatment of cytomegalovirus infection.
• Mitapivat (Pyrukynd, Agios Pharmaceuticals) to treat pyruvate kinase deficiency.
• Loncastuximab tesirine (Zynlonta, ADC Therapeutics) for a conditional marketing authorization to treat diffuse large B-cell lymphoma and high-grade B-cell lymphoma.