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orphan drug status

PharmaDeviceNews orphan drug status

Representative image: Corcept Therapeutics’ dazucorilant survival signal in amyotrophic lateral sclerosis highlights how ALS clinical trials are shifting attention from short-term functional scores to longer-term survival outcomes.

Pharma & Biotech

A failed ALS trial still showed a stunning survival signal. Can Corcept turn it into Phase 3 proof?

by Pallavi Madhiraju
May 3, 2026
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A failed ALS endpoint meets a striking survival signal. Corcept now faces the harder test: proving dazucorilant in Phase 3.

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POPULAR NEWS

Representative image of a clinician reviewing skeletal imaging, illustrating Mirum Pharmaceuticals and Incyte’s zilurgisertib Phase 2 progress in fibrodysplasia ossificans progressiva and the rare disease treatment questions facing regulators.

Pharma & Biotech

Why Mirum Pharmaceuticals and Incyte’s zilurgisertib data could reset expectations in FOP treatment

June 15, 2026

Niagen Bioscience’s NB4168 has secured U.S. FDA Rare Pediatric Disease and European orphan designations as the company advances the experimental ataxia telangiectasia therapy toward human trials. Representative image.

Pharma & Biotech

Niagen wins twin rare-disease designations, but can NB4168 turn nicotinamide riboside evidence into a medicine?

July 14, 2026

Representative image of biopharma laboratory research. Gilead Sciences and Merck’s latest Phase 3 updates highlight the promise of once-weekly HIV treatment with islatravir and lenacapavir, while raising fresh questions over Trodelvy’s lung cancer expansion strategy.

Pharma & Biotech

Gilead and Merck pipeline update reveals promise in HIV and pressure in oncology

June 9, 2026

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clinical trials Eli Lilly and Company European Medicines Agency FDA FDA approval immuno-oncology immunotherapy Inc. Johnson & Johnson medical devices oncology precision oncology U.S. Food and Drug Administration United States Food and Drug Administration US Food and Drug Administration

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