Rare diseases

R&D into orphan drugs is growing alongside the number of approved treatments, providing treatments for rare diseases that

Orphan drugs’ financial success raises questions

https://pharmaphorum.com/market-access-2/orphan-drugs-financial-success-raises-questions/

Patients/ Rare diseases/ Views & Analysis/ Views and analysis

Ring 20: Could the rare disease get left behind by next-generation gene sequencing?

mike.hammerton@pharmaphorum.com Exploring Rare Diseases, patient insights, Rare diseases, Ring 20 Research and Support, Ring Chromosome 20 syndrome 0 Comment

Ring Chromosome 20 Syndrome, or (R)20, is an ultra-rare form of epilepsy with a devastating impact – yet

Ring 20: Could the rare disease get left behind by next-generation gene sequencing?

https://pharmaphorum.com/views-analysis-patients/ring-20-could-the-rare-disease-get-left-behind-by-next-generation-gene-sequencing/

News/ Pfizer/ Rare diseases

Pfizer axes development of Duchenne muscular dystrophy antibody

Piotr Wnuk DMD, Duchenne, Duchenne muscular dystrophy, Pfizer, rare disease 0 Comment

Pfizer has axed trials of its domagrozumab antibody for Duchenne muscular dystrophy (DMD), after it failed against efficacy

Pfizer axes development of Duchenne muscular dystrophy antibody

https://pharmaphorum.com/news/duchenne-trials-axed-pfizer-dmd/

News/ Rare diseases

US regulators reject Akcea and Ionis rare disease drug

Piotr Wnuk Akcea Therapeutics, familial chylomicronemia syndrome, Ionis Pharmaceuticals, rare disease, Waylivra 0 Comment

The US Food and Drug Administration (FDA) rejected Akcea and Ionis’ application for the use of Waylivra in

US regulators reject Akcea and Ionis rare disease drug

https://pharmaphorum.com/news/us-regulators-reject-akcea-ionis-rare-disease-drug/

AstraZeneca/ Merck Inc/ News/ News/ News/ Oncology/ R&D/ Rare diseases/ Top stories/ UK & Europe

Neurofibromatosis treatment granted orphan status by EMA

Piotr Wnuk ASCO 2018, AstraZeneca, ema, genetic, Merck & Co, Neurofibromatosis, NF1, Oncology, Selumetinib 0 Comment

AstraZeneca and Merck & Co. announced the European Medicines Agency (EMA) has granted orphan designation to their codeveloped

Neurofibromatosis treatment granted orphan status by EMA

https://pharmaphorum.com/news/nf1-treatment-granted-orphan-status-ema/

Market Access/ News/ News/ News/ News/ Patients/ R&D/ Rare diseases

The first lipodystrophy treatment for European patients

Piotr Wnuk Lipodystrophy, Metreleptin, Myalept, Myalepta, Novelion Therapeutics 0 Comment

Novelion Therapeutics’ Myalepta (metreleptin) developed for the ultra-rare condition lipodystrophy has been approved by the European Commission, giving

The first lipodystrophy treatment for European patients

https://pharmaphorum.com/news/the-first-lipodystrophy-treatment-for-european-patients/

News/ News/ R&D/ Rare diseases/ Transversal Categories

Shionogi’s Mulpleta gets FDA green light for patients with liver disease

Joy Persaud FDA approval, lusutrombopag, Shionogi, thrombocytopenia 0 Comment

The US drug regulator has approved Mulpleta (lusutrombopag) as a treatment for thrombocytopenia in certain patients with chronic

Shionogi’s Mulpleta gets FDA green light for patients with liver disease

https://pharmaphorum.com/news/shionogis-mulpleta-gets-fda-green-light-for-patients-with-liver-disease/

News/ Rare diseases/ US

Alexion hastens review of successor to rare disease blockbuster Soliris

Richard Staines Alexion, Amgen, Rare diseases 0 Comment

Alexion’s rare disease drug Soliris is generating blockbuster-level sales – but the US biotech is rushing through development

Alexion hastens review of successor to rare disease blockbuster Soliris

https://pharmaphorum.com/news/alexion-hastens-review-of-successor-to-rare-disease-blockbuster-soliris/

Market Access/ News/ News/ Rare diseases/ Top stories/ UK & Europe

Patients call for end to two-year row over CF drug funding

Richard Staines cystic fibrosis, market access, NICE, Rare diseases, Vertex 0 Comment

UK patients are stepping up efforts to resolve the “heartbreaking” row that is denying their families access to

Patients call for end to two-year row over CF drug funding

https://pharmaphorum.com/news/patients-call-for-end-to-two-year-row-over-cf-drug-funding/

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