Nacuity Pharmaceuticals Inc. and Arctic Therapeutics LLC have entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of NPI-001 (N-acetylcysteine amide, NACA) in Iceland for the treatment of hereditary cystatin C amyloid angiopathy (HCCAA).
Brightinsight Inc. and UCB SA struck a deal to jointly develop a digital disease management solution for patients with rare diseases. The companies will start with a mobile app for myasthenia gravis (MG) built on Brightinsight’s platform. UCB joins CSL Behring, Novo Nordisk A/S, Sanofi SA and Roche AG in partnering with Brightinsight on digital disease management.
The ultra-rare skin disease X-linked hypohidrotic ectodermal dysplasia (XLHED) can be a heavy burden for children, affecting structures in the skin including sweat glands, respiratory glands, and hair as well as teeth. Though lacking a specific treatment to date, a pivotal clinical trial planned by the Geneva-based nonprofit Esperare Foundation and Pierre Fabre SA could now help bring one about.
LONDON – The industry is expressing divergent views of changes to how the U.K. health technology assessment agency, the National Institute for Health and Care Excellence (NICE), will in the future select what products to assess and the methods and processes it will use to carry out its evaluations.
LONDON – The industry is expressing divergent views of changes to how the U.K. health technology assessment agency, the National Institute for Health and Care Excellence (NICE), will in the future select what products to assess and the methods and processes it will use to carry out its evaluations.
LONDON – A pilot study has shown that whole genome sequencing can pinpoint the genetic causes of rare diseases, even in people who had previously not been given a diagnosis after undergoing sequencing of their protein coding exome.
Centogene NV and Twist Bioscience Corp. are linking up to develop custom assay kits for rare disease testing. The companies said the collaboration aims to make genetic tests more accessible to rare disease patients. Financial details were not disclosed. Centogene, which dubs itself the “rare disease company” is positioning to be a frontrunner in the testing market. The global rare disease genetic testing market size was valued at $812.3 million in 2019 and is projected to register a CAGR of 10.9% from 2020 to 2027.
Astrazeneca plc’s Alexion rare diseases unit has taken an option to buy all remaining equity in Caelum Biosciences Inc., adding a rare disease drug to its pipeline in a deal worth $500 million. At the center of the deal is CAEL-101, a potentially first-in-class monoclonal antibody designed to tackle the toxic amyloid deposits that build up in organs of people with the rare disease light chain amyloidosis.
In one of the biggest collaboration deals of the year, Shape Therapeutics Inc. entered a collaboration and license agreement with Roche Holding AG to develop gene therapies for targets in areas that include Alzheimer’s disease, Parkinson’s disease and rare diseases. Seattle-based Shape is eligible to receive an initial payment, development, regulatory and sales milestone payments that could exceed $3 billion in aggregate value.
Albireo Pharma Inc.’s Bylvay (odevixibat) becomes the first therapy to hit the commercial market for rare genetic liver disease progressive familial intrahepatic cholestasis (PFIC). A day after the European Commission approved the drug, the FDA gave the nod to the ileal bile acid transport inhibitor on its July 20 PDUFA date following a priority review.
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