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orphen drugs

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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

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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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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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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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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Infographic: Rare Diseases in China

How rare are rare diseases in China? Download this infographic to find out more and how we can offer guidance for pharma companies to consider.

Research Partnership

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Landscape review reveals £1.1bn of UK funding for rare diseases

The report focuses on future funding and needs for those with rare diseases

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Clinigen launches educational programme for rare disease patients and organisations

The NaviGATE programme will aim to empower patients and provide navigational support

regeneron headquarters

Regeneron’s Veopoz granted FDA approval for ultra-rare immune disease

Fewer than ten patients in the US have been diagnosed with CHAPLE disease

Biogen Idec building

Biogen to acquire Reata for approximately $7.3bn

The deal gives Biogen access to a recently launched therapy for Friedreich's ataxia

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AstraZeneca’s rare disease unit to acquire Pfizer’s early-stage gene therapies for $1bn

The transaction could potentially see staff associated with the portfolio move to Alexion

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