Rare Disease

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.

News

Chiesi expands rare disease portfolio with Amryt Pharma acqu...

Biopharma and healthcare group Chiesi Farmaceutici S.p.A.

News

Takeda claims first phase 3 win in rare disease cTTP

Takeda has reported positive results from a phase 3 trial of its experimental therapy TAK-755 for ultra-rare disease congenital thrombotic thrombocytopenic purpura (cTTP), setting up regula

News

Atara claims first world approval for off-the-shelf T-cell t...

Atara Biotherapeutics has become the first biotech to win regulatory approval for a T-cell therapy based on donated cells, rather than relying on cells harvested from patients and processed

News

CHMP backs Amicus' therapy for Pompe disease

Amicus Therapeutics could be just weeks away from getting EU approval for its new Pompe disease therapy Pombiliti after getting a positive opinion on the drug from the EMA's human medicines

Views & Analysis

How eligible patient numbers affect price of rare disease dr...

Having recently assessed the relationship between drug price and prevalence of non-oncology rare diseases, CRA’s Life Sciences Practice team explores in greater depth the situation for thes

Acquisitive Ipsen builds again with $952m Albireo buy

Chiesi expands rare disease portfolio with Amryt Pharma acqu...

Takeda claims first phase 3 win in rare disease cTTP

Atara claims first world approval for off-the-shelf T-cell t...

CHMP backs Amicus' therapy for Pompe disease

How eligible patient numbers affect price of rare disease dr...

Lilly grabs first-ever FDA nod for a sleep apnoea drug

The AI effect in pharma commercial strategy: From data to re...

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