The first treatment for the rare neurodegenerative disorder Friedreich’s ataxia relied on a single placebo-controlled efficacy trial.
Reata Pharmaceuticals, Inc. notched its first approval from its pipeline of drug candidates that activate the transcription factor Nrf2 when the US FDA approved Skyclarys (omaveloxolone) on 28 February 2023 as the first treatment for the Friedreich’s ataxia, an ultra-rare neurodegenerative disorder that is typically diagnosed in adolescence.
Reata emphasized clarity on FDA expectations for confirmatory evidence during the clinical development of omaveloxolone, which the company planned to...
Upcoming FDA user fee goal dates include novel products from a busy lung cancer pipeline, a single-dose passive immunization against RSV for infants, what could be the first oral HAE acute treatment, and a first-in-class HAE preventive antibody.
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.
In the infrequent cases when EU authorization precedes US FDA approvals of new products, biologics manufacturing issues are usually to blame, a Pink Sheet analysis found.
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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.
