Rare Disease

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Ipsen's rare disease drug palovarotene knocked back in EU

Ipsen has suffered a setback in its marathon effort to bring palovarotene to market for ultra rare disease fibrodysplasia ossificans progressiva (FOP), after EU advisors recommended against

Market Access

Innovative Medicines Fund and the opportunity for ICSs-indus...

In June 2022, NHS England (NHSE) and the National Institute for Health and Care Excellence (NICE) launched the Innovative Medicines Fund (IMF).

News

NICE backs Alnylam's Amvuttra for rare form of amyloidosis

Alnylam's gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related (ATTR) amyloidosis by NICE, paving the way for the drug to be made available

News

Blockbusters in waiting: Clarivate's drugs to watch in 2023

New drugs for immunological and inflammatory diseases feature prominently among new product launches this year that could top $1 billion in annual sales by 2027 or be clinical "game changer

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Astellas licenses companion drug for Pompe gene therapy

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe

News

Acquisitive Ipsen builds again with $952m Albireo buy

French drugmaker Ipsen has continued a recent spate of deal-making with a $42-per-share offer to buy US biotech Albireo and its rare disease therapy Bylvay.