Regulatory Updates

In February 2022, the FDA had issued a complete response letter in order to inspect a new third-party packaging and labeling facility.

The FDA has set a PDUFA date of Jan. 29, 2023, but Merck indicated additional data may be provided to regulators that extends this date.

If approved, beti-cel will be the first potentially curative gene therapy for people with beta-thalassemia who require regular red blood cell transfusions. The PDUFA date is Aug. 19, 2022.

If approved, elivaldogene autotemcel will be the first gene therapy to address the underlying genetic cause of cerebral adrenoleukodystrophy, a rare disease that affects young boys.

Efanesoctocog alfa is a new class of factor VIII therapy with once-weekly prophylactic dosing. The application for approval expected to be filed mid year.

Novavax’s COVID-19 vaccine demonstrated 90.4% efficacy with a low number of serious and severe adverse events, including myocarditis.

Dupixent is the first biologic medicine approved to treat moderate-to-severe atopic dermatitis from infancy through adulthood.

Riabni is now approved to treat all available Rituxan indications.

Reblozyl was being reviewed to treat anemia in adults with non-transfusion dependent beta thalassemia, an inherited blood disorder. BMS indicated it couldn’t appropriately address FDA’s questions about risk-benefit.

The FDA has extended review time to allow for review of additional analyses of data from the company’s clinical studies. The new PDUFA date is Sept. 29, 2022.

The FDA is asking for additional data to support effectiveness of pegzilarginase to treat a rare metabolic disease. If approved, pegzilarginase would be the first treatment for ARG1 deficiency.

The regulatory agency indicated there is an issue surrounding the protocol design.

If approved, Dupixent would be the first and only medicine in the United States specifically indicated to treat prurigo nodularis, or persistent itch with thick skin lesions. The PDUFA target action date for the FDA decision is Sept. 30, 2022.

The FDA indicated the risk of death outweighs the benefits of Ukoniq, which was approved in February 2021 to treat specific lymphomas.

Evrysdi is now approved to treat spinal muscular atrophy in children and adults of all ages.

Opdivo-based treatments are now approved for five indications in upper gastroesophageal cancers.

Kymriah is now approved in three indications and is the only CAR-T cell therapy approved in both adult and pediatric settings.

This is the fifth biosimilar of Neulasta and the third biosimilar from Amneal to receive FDA approval.

The FDA has assigned a Prescription Drug User Fee Act target action date of Nov. 30, 2022. Regulators indicated they plan to hold an advisory committee meeting to discuss the application.

Tibsovo is the first therapy targeting cancer metabolism for patients with newly diagnosed IDH1-mutated acute myeloid leukemia.

Deficiencies at the contract manufacturing company are, for the second time, holding up approval of VP-102 to treat molluscum contagiosum, a mild skin infection.

If approved, etranacogene dezaparvovec would be the first gene therapy for patients with hemophilia B. An FDA decision is expected in November 2022.

Vtama is approved to treat adults with mild, moderate, and severe psoriasis. It is expected to be available in the first week of June 2022.

Tyvaso DPI is a dry inhalation powder and new inhalation device to treat patients with pulmonary arterial hypertension, a life-threatening disease. It is expected to be available in June 2022.

The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date for the first quarter of 2023 for zavegepant, a nasal spray for the acute treatment of patients with migraine.

The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of Nov. 28, 2022.

Dupixent is the first medicine specifically to treat eosinophilic esophagitis, an inflammatory condition that causes the esophagus to narrow and makes it difficult to eat.

ABBV-951 is a combination of foscarbidopa and foslevodopa, prodrugs of carbidopa and levodopa, that is designed to provide 24-hour, continuous subcutaneous delivery.

Bimekizumab, now called Bimzelx, is a monoclonal antibody being reviewed as treatment for adults with moderate-to-severe plaque psoriasis.

While regulatory flexibility is important for drugs for rare diseases, investigators are concerned the trend toward surrogate endpoints decreases confidence that new drugs can improve patient outcomes.