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Fda Rejection

All articles tagged with #fda rejection

health-biotechnology12 days ago•3 min saved

FDA rejects Disc Medicine's bitopertin over surrogate-endpoint concerns

The FDA declined Disc Medicine’s bitopertin for erythropoietic protoporphyria, citing that the phase 2 trials used a surrogate endpoint (a ~40% drop in PPIX) whose link to meaningful clinical benefit—sun tolerance—remains unproven. The company plans to press on with the APOLLO phase 3 trial, which uses sun-exposure outcomes, while the broader regulatory environment and the CNPV priority voucher program continue to draw scrutiny.

via Fierce Biotech|

#bitopertin #disc-medicine #erythropoietic-protoporphyria

healthcare6 months ago•1 min saved

Stealth BioResubmits Rare Disease Drug Application Amid FDA Challenges

Stealth BioTherapeutics disclosed an FDA rejection letter to rally support for its experimental drug for Barth syndrome, a rare disease affecting about 150 people in the U.S., amid ongoing struggles to meet regulatory requirements and concerns over the company's future.

via statnews.com|

#barth-syndrome #fda-rejection #healthcare

business7 months ago•2 min saved

Replimune Shares Drop 75% After FDA Skin Cancer Drug Rejection

Replimune's stock plummeted 77.2% after the FDA unexpectedly rejected its experimental melanoma drug, citing insufficient data from midstage studies, which surprised investors and analysts who had anticipated approval based on positive preliminary results.

via Investor's Business Daily|

#biotech #business #fda-rejection

business7 months ago•3 min saved

Capricor Shares Drop After FDA Rejection of DMD Cell Therapy

Shares of Capricor Therapeutics dropped 33% after the FDA rejected its experimental drug deramiocel for treating cardiomyopathy in Duchenne muscular dystrophy, citing insufficient evidence of effectiveness and issues in manufacturing documentation. The company plans to continue testing with upcoming results from ongoing studies that could support future approval efforts.

via Investor's Business Daily|

#business #capricor-therapeutics #cardiomyopathy

disasters2 years ago•6 min saved

"FDA Rejects Epinephrine Nasal Spray and Taylor Swift Vault Search Glitches: Morning Rundown Highlights"

Maui firefighters recount their efforts to battle the devastating blaze that destroyed the town of Lahaina, Hawaii, facing dangerous conditions and making life-saving decisions. The FDA rejects Neffy, a needle-free alternative to EpiPens, requesting additional data from drugmaker ARS Pharmaceuticals. Ukrainian President Volodymyr Zelenskyy appeals for international support in holding the line against Moscow's aggression. Republican divisions in the House increase the likelihood of a government shutdown. Fans searching for Taylor Swift's unreleased songs encounter glitches on Google. Olive oil prices surge, leading to thefts.

via NBC News|

#disasters #fda-rejection #maui-firefighters