Agency questions whether post hoc analyses and use of natural history subjects as an external control group in a Phase III study support effectiveness of palovarotene; cites apparent increased incidence of flare-up events as a key safety issue.
The US Food and Drug Administration expressed uncertainty about whether a Phase III study of Ipsen SA’s palovarotene demonstrates efficacy for treatment of patients with fibrodysplasia ossificans progressiva since the study failed its prespecified primary efficacy analysis and the new drug application relies on post hoc analyses to support a showing of efficacy.
The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee is to consider Ipsen’s application at its 28 June meeting. In a
Advisory committee for Capricor’s deriamocel planned, regenerative medicine advanced therapy designations proliferate for osteoarthritis, and the ranks of dual RMAT/breakthrough therapy designation holders grow.
The 31 May goal date for Moderna’s next-generation COVID-19 vaccine looms over the month, but the US FDA's decision lineup includes three more novel agents, including AbbVie's Teliso-V antibody-drug conjugate, as well as supplemental indications like GSK's bid for Nucala in COPD.
Antabio’s next-generation, broad-spectrum antibacterial, MEM-ANT3310, has made it onto the European Medicines Agency’s priority medicines scheme. The company says its product is designed to make a significant impact on the growing problem of antimicrobial resistance.
Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.
Stay up to date on regulatory guidelines from around the world with the Pink Sheet’s Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.
Organizational turmoil has not yet affected reviews of novel agent applications, with Q1 2025 approvals coming in low, but the first quarter share of the entire year is variable.
This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
