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Novartis to acquire Excellergy in deal worth up to $2bn

Exl-111, a potential treatment included in the deal, is a central factor in many allergic diseases

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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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Scotland becomes first part of UK to screen all newborn babies for SMA

The screening is part of a two-year evaluation funded by the Scottish government and Novartis

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Novartis agrees on potential $3bn deal to buy Synnovations’s breast cancer treatment

The oral drug is currently being evaluated for breast cancer and other advanced solid tumours in a phase 1/2 study

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Lundbeck completes patient randomisation for phase 3 trial for multiple system atrophy treatment

The rare neurodegenerative disorder has no cure and no approved treatments

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US FDA launches ultra-rare diseases framework

The draft guidance specifically discusses genome editing and RNA-based therapies

phthalmologist, optometrist checking child, little girl vision looking for problems and caring for eye

Why patient engagement must come first in rare disease trials

Explore why patient engagement is the cornerstone of successful rare disease trials, emphasising the importance of designing studies around the realities of patients' lives to build trust and improve outcomes.

Cuttsy + Cuttsy

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New Cuttsy+Cuttsy research shows why site support matters more than ever

Cuttsy+Cuttsy's upcoming report, 'The site factor: Where protocol meets practice in clinical trials', delves into the critical role of site staff in clinical research, offering insights into their daily challenges,...

Cuttsy + Cuttsy

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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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AstraZeneca and Daiichi Sankyo’s Datroway given FDA Priority Review for breast cancer treatment

Approximately 70% of metastatic triple-negative breast cancer patients are ineligible for immunotherapy, creating an unmet medical need

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Johnson & Johnson’s Darzalex Faspro regimen receives FDA approval for multiple myeloma

Multiple myeloma has an average five-year survival rate of 59.8% and is currently incurable

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