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Myalept

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LEO Pharma to acquire Replay gene therapy platform

The acquisition will advance innovation for patients with rare skin diseases

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FDA gives Rare Paediatric Disease designation to Satellite Bio for urea cycle disorders

The mortality rates of UCDs exceeds 25%, with no curative treatment available for newborns

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AstraZeneca reports positive phase 3 data for tozorakimab in COPD

COPD affects nearly 400 million people globally and is the third leading cause of death worldwide

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Shionogi starts phase 2 clinical trial for Pompe Disease

The rare disease affects around one in every 22,000 people worldwide

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PharmaQuest returns: EPA perspectives, through a rare disease lens

By Lottie Sequerra and Emily Morton-West

Amiculum

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BMS’ Opdivo gets expanded FDA and EU approval for Hodgkin lymphoma

This type of cancer is the most common one diagnosed in teenagers

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AstraZeneca’s Imfinzi approved in EU for early gastric and gastro-oesophageal cancers

This is the first and only perioperative immunotherapy approved to treat early gastric cancers

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US FDA launches ultra-rare diseases framework

The draft guidance specifically discusses genome editing and RNA-based therapies

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PureTech’s deupirfenidone gets FDA and EC Orphan Drug Designations for idiopathic pulmonary fibrosis

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or fewer than five in 10,000 individuals in the European Union

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Diabeloop’s DBLG2 receives FDA and CE clearance for automated insulin delivery without meal input

While a lack of meal announcements can lead to lower glycaemic control, it reduces the mental burden for those with type 1 diabetes

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UCB’s Kygevvi receives positive CHMP opinion for thymidine kinase 2 deficiency

TK2d is an extremely rare disease characterised by severe and progressive muscle weakness

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Sanofi announces positive results for venglustat in type 3 Gaucher disease

There are currently no approved treatments for the neurological symptoms of this disease

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