Rare diseases

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Norgine's chief executive Janneke van der Kamp

Norgine on a growth charge again with Theravia takeover deal

Netherlands-based pharma group Norgine has expanded its rare disease portfolio with the acquisition of France's Theravia for an undisclosed sum.

News

Cheaper Soliris biosimilars reach the US market

The first biosimilars of AstraZeneca/Alexion's blockbuster complement C5 inhibitor Soliris have been launched in the US at a discount to the brand.

News

Uplizna is first drug FDA-cleared for rare disease IgG4-RD

Amgen has bagged a second indication for its B cell-depleting therapy Uplizna after the FDA cleared it for rare inflammatory disease IgG4-RD.

News

FDA clears first treatment for Prader-Willi syndrome

Soleno Therapeutics' Vykat XR has become the first FDA-approved treatment for excessive eating in rare disorder Prader-Willi syndrome.

News

Fabhalta is first FDA-approved therapy for rare disease C3G

Novartis' oral complement inhibitor Fabhalta has become the first approved treatment for rare kidney disease C3G as it chases a $3bn sales target.

News

Third indication beckons for Amgen's Uplizna

Amgen is preparing to file a third indication for Uplizna, as a twice-yearly therapy for generalised myasthenia gravis, following new phase 3 data.

Norgine on a growth charge again with Theravia takeover deal

Cheaper Soliris biosimilars reach the US market

Uplizna is first drug FDA-cleared for rare disease IgG4-RD

FDA clears first treatment for Prader-Willi syndrome

Fabhalta is first FDA-approved therapy for rare disease C3G

Third indication beckons for Amgen's Uplizna

Prevention for a healthier, more productive society

Health Trends 2025: Reimagining What's Possible

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