Articles related to FDA

CheckRare provides a list of the orphaned drugs that dominate the US pharmaceutical industry to start 2022. Orphan drugs are defined as those medications that remain commercially undeveloped because they’ve been deemed to have limited potential for profitability. However, nearly half of all novel medications approved by the FDA are orphan drugs.

For patients being prescribed isotretinoin, there used to be three risk categories: women of reproductive potential, women not of reproductive potential, and males. The new risk classifications are two: those who can become pregnant and those who cannot. This change was based on stakeholder feedback that gender-specific language can be a barrier to access for patients who do not identify with the previous gender-specific patient categories.

The U.S. Food and Drug Administration (FDA) held a two-day public meeting to discuss managing the potential side effects of gene therapies following the death of a boy with a rare neuromuscular disease. High doses of gene therapies are known to cause liver damage, and recent studies have also found a potential long-term cancer risk in mice and dogs treated with gene therapies.

The FDA has required that Amgen compare its approved KRAS-targeted therapy sotorasib (Lumakras) 960-mg dose with a 240-mg dose as one of its accelerated approval post-marketing requirements, a paradigm-shifting event that signals new scrutiny around determining the labeled dose for targeted oncology drugs, which still follow outdated maximum tolerated dose (MTD) principles in cytotoxic agents.

The investigational agent – known as REGEN-COV (casirivimab and imdevimab) – was previously granted the Emergency Use Authorization (EUA) for the treatment of mild to moderate COVID-19. Now, those who are at high risk for progression to severe COVID-19, including hospitalization or death, have an option.

The additional approval expands immunization coverage to the most at-risk group for acquiring shingles – the immunocompromised. It also shortens the time to the second dose in the 2-dose Shingrix schedule.  Decision support based on the Zoster Efficacy Study in Patients Undergoing HSCT (ZOE-HSCT) phase III trial (JAMA).