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Molybdenum cofactor deficiency (MoCD) type A, an ultra-rare metabolic disorder causing intractable seizures, brain injury and death, now has a world-first treatment in Nulibry (fosdenopterin), a new I.V. therapy developed by Bridgebio Pharma Inc. subsidiary Origin Biosciences Inc. The agency's priority review, supported by its orphan, breakthrough and rare pediatric disease programs, also yielded a priority review voucher (PRV) for Origin.
LONDON – Exosome specialist Evox Therapeutics Ltd. has raised $95.4 million in a series C round that will enable it to progress a number of rare disease programs into the clinic, with the lead due to enter phase I at the beginning of next year.
Avrobio Inc. said Feb. 8 that the first person dosed with AVR-RD-01, an investigational ex vivo lentiviral gene therapy from its upgraded manufacturing platform, Plato, experienced a complete clearance of the toxic substrate lyso-Gb3 in a kidney biopsy.
Seeking to accelerate the company's long-term growth through an expansion of its rare disease pipeline, Horizon Therapeutics plc said Feb. 1 it will buy Viela Bio Inc. for $3.05 billion, or $53 per share (NASDAQ:VIE). Once closed, the deal would add a new commercial-stage asset, Uplizna (inebilizumab), to Horizon's portfolio alongside its current lead products, Tepezza (teprotumumab) for the treatment of thyroid eye disease and Krystexxa (pegloticase) for uncontrolled gout. Dublin-based Horizon, which had $2.08 billion in cash at the end of 2020, borrowed $1.3 billion to help finance the transaction. Astrazeneca plc also enabled the deal by agreeing to divest its 26.7% share in Viela Bio for a profit of between $760 million and $780 million.
Using an adenine base editor, researchers at the U.S. NIH, Vanderbilt University Medical Center and Harvard University have succeeded in treating the premature aging syndrome Hutchinson-Guilford progeria syndrome (progeria) in mice, extending the animals’ lifespan and preventing much of the vascular damage that is typically the cause of death in children with progeria.
Apellis Pharmaceuticals Inc. could receive up to $1.25 billion from Swedish Orphan Biovitrum (Sobi) AB in their collaboration to develop systemic pegcetacoplan, a C3 therapy for treating several rare diseases in hematology, nephrology and neurology.
BEIJING – Canbridge Pharmaceuticals Inc. last week won marketing approval in China for its first rare disease drug, the mucopolysaccharidosis II therapy Hunterase (idursulfase beta injection).
Millendo Therapeutics Inc. in April decided to scrap work altogether with livoletide in Prader-Willi syndrome after a phase IIb study fell short of statistical significance, but hope remains alive for – among others in the space – Soleno Therapeutics Inc., another recent newsmaker with once-daily diazoxide choline controlled-release tablets in PWS.
DUBLIN – Ipsen SA is on track for an NDA filing for palovarotene in fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease characterized by the gradual replacement of skeletal muscle and connective tissue with bone, following an interim analysis of phase III data which indicates that the drug may have a substantial effect on the disease process.
A third child with X-linked myotubular myopathy (XLMTM) has died after receiving the higher of two doses of an experimental gene therapy for the rare disease under development at Audentes Therapeutics Inc. Preliminary findings indicate that the immediate cause of death was gastrointestinal bleeding, Audentes said. The trial, testing AT-132 (resamirigene bilparvovec), was already on an FDA clinical hold.
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