News round-up for 17-21 February by DDW Senior Digital Content Editor Diana Spencer.

Following the news of cuts to medical research funding from the National Institutes of Health (NIH), this week we focus on some positive drug discovery news from North America, including FDA priority review for a number of innovative therapeutics and a new US centre for rare disease research.

The top stories:

Novartis receives Health Canada approval for Fabhalta

Health Canada has granted a Notice of Compliance for Fabhalta (iptacopan capsules), the first oral monotherapy treatment for adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

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New US centre for rare diseases to promote drug research

Washington University School of Medicine in St Louis has launched the Center for Rare, Undiagnosed and Genetic Diseases, supported by an $8.5 million grant from Children’s Discovery Institute (CDI), a partnership with St Louis Children’s Hospital, St Louis Children’s Hospital Foundation and WashU Medicine.

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Dupixent for bullous pemphigoid under FDA priority review

The US Food and Drug Administration (FDA) has accepted for priority review the supplemental biologics license application (sBLA) for Dupixent (dupilumab) to treat adults with bullous pemphigoid (BP).

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Targeted medicine for NSCLC receives FDA Priority Review

The US Food and Drug Administration (FDA) has granted Priority Review to Boehringer Ingelheim’s new drug application for zongertinib for unresectable or metastatic non-small cell lung cancer (NSCLC).

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Further FDA support for Parkinson’s gene therapy

AskBio’s investigational gene therapy AB-1005 for the treatment of Parkinson’s disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the US Food and Drug Administration (FDA).

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