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FDA delays approval decision for Biohaven rare disease drug

BioPharma Drive: Drug Pricing

Shares of the biotech fell around 15% on news the FDA is not only taking longer to review the drug, but will assemble an outside group of advisers to evaluate it as well.

FDA 248
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FDA OKs first blood test to aid Alzheimer’s diagnosis

BioPharma Drive: Drug Pricing

The FDA cleared the test for early detection of amyloid plaques associated with Alzheimer’s in people aged 55 years and older with signs and symptoms of the disease.

FDA 162
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FDA Advisors Say New Gene Therapy for Sickle Cell Disease, Exa-cel, is Safe

Drugs.com

1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. The FDA had already decided that the. WEDNESDAY, Nov. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December.

Therapies 263
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Novartis gets FDA approval of closely watched rare disease drug

BioPharma Drive: Drug Pricing

The pharma is developing Fabhalta, now cleared for paroxysmal nocturnal hemoglobinuria, for several other rare, complement-driven diseases.

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Verve gets FDA green light to run base editing study in US

BioPharma Drive: Drug Pricing

since late last year as the FDA sought more details on Verve’s in vivo treatment for heart disease. The trial, which is ongoing in the U.K. and New Zealand, has been on hold in the U.S.

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FDA clears CSL’s swelling disease drug; Regeneron loses 23andMe auction

BioPharma Drive: Drug Pricing

The agency cleared CSL’s hereditary angioedema drug days after “resource constraints” delayed a rival medicine. Elsewhere, Intellia revealed new data and Lilly beefed up an online service offering access to Zepbound.

Disease 147
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FDA lifts hold on PTC Huntington’s disease trial

BioPharma Drive: Drug Pricing

The agency has lifted a partial trial suspension based on one-year data showing PTC’s pill suppressed a key protein associated with the disorder.

Trials 312