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Novartis shares positive phase 3 results for Lutathera in neuroendocrine tumours

The incidence of neuroendocrine tumours has increased over the past several decades

- PMLiVE

MHRA approves UCB’s Zilbrysq as generalised myasthenia gravis add-on therapy

The autoimmune disease has a global prevalence of 100 to 350 cases per every one million people

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Takeda’s HyQvia granted FDA approval to treat rare neuromuscular disorder CIDP

Approximately five to seven people per 100,000 in the US are affected by the disorder

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Novartis shares positive phase 3 results for Scemblix in chronic myeloid leukaemia

More than 6,300 people are diagnosed with the disease every year in Europe

On a journey to diverse, inclusive, and more efficient rare disease trials

Although orphan drugs represent a sizeable portion of new drug approvals, most rare diseases still lack effective treatment. Even more so than for other conditions, rare disease management contains large care...

Impetus Digital

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Novartis and Voyager enter gene therapy partnership worth over $1.2bn

The companies will advance candidates for Huntington’s disease and spinal muscular atrophy

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AstraZeneca and Ionis’ Wainua granted FDA approval for rare disease ATTRv-PN

ATTRv-PN leads to peripheral nerve damage with motor disability within five years of diagnosis

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Novartis’ Fabhalta approved by FDA as first oral monotherapy for rare blood disease PNH

The rare blood disease is estimated to affect only ten to 20 people per million worldwide

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Merck to acquire Caraway Therapeutics in a deal worth up to $610m

The deal gives Merck access to a pipeline of therapeutics for neurodegenerative and rare diseases

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Novartis gains global rights to Legend’s CAR-T cell therapies in deal worth over $1bn

Novartis can apply its T-Charge platform to the manufacturing process of the therapies

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NICE recommends Chiesi’s alpha-mannosidosis enzyme replacement therapy

Alpha-mannosidosis, an extremely rare inherited condition characterised by a broad range of impairments, affects approximately 25 people in England

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Novartis’ Cosentyx receives FDA approval for hidradenitis suppurativa

The decision makes Cosentyx the first biologic treatment approved for HS in nearly a decade

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