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Biogen receives EC approval for Skyclarys in Friedreich’s ataxia patients

The company gained access to the drug through its $7.3bn acquisition of Reata Pharmaceuticals

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EC approves Vertex/CRISPR’s Casgevy gene therapy for inherited blood disorders

There are now more than 8,000 patients who are potentially eligible for treatment with Casgevy

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Sanofi presents positive results for Pompe disease enzyme replacement therapy

The rare genetic disorder is estimated to affect around one in every 40,000 people in the UK

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Takeda’s HyQvia receives EC approval for rare neuromuscular disorder CIDP

HyQvia can now be used as a maintenance therapy for patients of all ages with CIDP

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GSK’s Omjjara approved by EC to treat myelofibrosis patients with anaemia

About 40% of myelofibrosis patients have moderate-to-severe anaemia at the time of diagnosis

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Sanofi/Regeneron’s Dupixent approved by FDA for younger eosinophilic oesophagitis patients

Around 21,000 children aged under 12 years in the US are being treated for the inflammatory disease

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Incisionless device to transform diabetes, liver disease and severe obesity treatment

ForePass replicates common invasive metabolic surgeries without making any incisions

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Sanofi expands rare disease pipeline with $2.2bn Inhibrx acquisition

The deal includes a mid-stage candidate for alpha-1 antitrypsin deficiency

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Sanofi/Regeneron’s Dupixent gets US label update for atopic dermatitis

Dupixent’s label now includes data supporting its use in hand and foot atopic dermatitis

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EC opens participation to new alliance addressing critical drug shortages

The Critical Medicines Alliance is expected to publish its first advice by autumn 2024

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Roche’s subcutaneous Tecentriq granted EC approval for multiple cancer types

The formulation reduces treatment time by around 80% compared to intravenous infusion

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Pfizer’s Talzenna combination receives EC approval for metastatic prostate cancer

Approximately 470,000 new cases of prostate cancer were reported in Europe in 2020

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