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tag amyotrophic-lateral-sclerosis

Amylyx ALS drug draws criticism over $158,000 price tag

pharmaphorum

OCTOBER 3, 2022

Amylyx Pharmaceuticals’ Relyvrio – a new treatment for amyotrophic lateral sclerosis (ALS) approved by the FDA on Thursday, after review of the data from its phase 2 trial – was the next day set at a list price of $158,000 per year in the US, sparking outcry. ALS affects over 30,000 people in the US.

Insurance

Insurance Prescription FDA Patients

Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

Pharmaceutical Technology

JUNE 29, 2022

The US Food and Drug Administration (FDA) put a high-profile bluebird bio trial for sickle cell disease on partial clinical hold, and advisory panels deliberated over decisions involving gene therapies for amyotrophic lateral sclerosis (ALS), cerebral adrenoleukodystrophy (CALD), and beta-thalassemia.

Food and Drug Administration

Food and Drug Administration FDA Marketing Pharmaceutical

Amylyx ALS drug draws criticism over $158,000 price tag

Rare Disease Spotlight – tracing the rise of orphan drug designations over almost 40 years

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