Politics and Policy

If approved, Qulipta would be the first oral calcitonin gene-related peptide receptor antagonist with a broad preventive treatment of both chronic and episodic migraine.

Trial results showed overall survival was shorter compared with chemotherapy. The move doesn’t affect other indications.

Sierra Oncology is seeking approval of momelotinib as a treatment for patients with myelofibrosis, a bone marrow cancer.

Committee members said the data from the two trials submitted didn’t support efficacy of Nuplazid in Alzheimer’s patients. The PDUFA target date is Aug. 4, 2022.

On June 15, 2022, the FDA’s Vaccines and Related Biological Products Advisory Committee unanimously voted in favor of both vaccines.

This is the third approval for Skyrizi, which also treats psoriatic arthritis and plaque psoriasis.

The FDA also issued a complete response letter for Imcivree for use in patients with Alstrom syndrome, another genetic rare disease that can cause obesity.

The committee recommended Moderna’s and Pfizer’s COVID-19 vaccines were recommended for children 6 months to five years, as well as Moderna’s vaccine for children ages 7 to 16.

But investigators found that U.S. approvals often come before publication of results.

Amvuttra is expected to launch in July, and the developer, Alnylam, plans to make this available through value-based agreements.

Olumiant is the first systemic therapy available to treat alopecia.

The FDA needs time to review additional clinical data submitted by BeiGene for the leukemia/lymphoma indication. The new date is Jan. 20, 2023.

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

Researchers analyzed studies from several countries and found a link between improved transitional care for those in long-term care facilities and a lower risk of a return trip to the hospital.

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.