Rare Diseases
The Swiss pharma acquired Kate Therapeutics for up to $1.1bn, gaining preclinical gene therapies for neuromuscular conditions and novel platform technology.
But differences in trial design means it is hard to handicap GSK’s product against Mirum’s volixibat.
The company is trading below cash after admitting that an adverse event in a Rett syndrome trial left a patient critically ill.
The company has reached agreement with the US FDA on a pivotal trial for RGX-202 through an accelerated pathway.
With its data monitoring committee saying a Phase III trial of a drug-device combo product in pediatric myopia was not on track for success, Eyenovia will end the study and consider its options.
The MEK inhibitor, already approved for pediatric patients with the rare condition neurofibromatosis type 1, showed benefits for adults in a Phase III trial.
Despite a successful Phase III study of ALXN-1840 for the inherited disorder that prevents the liver from filtering copper out of the body, the UK major's rare disease division Alexion pulled the plug on the program last summer. However, Illinois-based biotech Monopar is confident in its potential.
Ocuphire will shift toward gene therapy for inherited retinal degeneration via a merger that will take Opus Genetics public. The firm will also be responsible for one of Ocuphire’s pipeline projects.
CEO of Sumitomo Pharma America unit, Adele M. Gulfo, draws from experiences working across companies including Pfizer, to advise on go-to-market strategies for a portfolio across primary care to rare disease.
Sanofi and Orano Med have kicked off a joint venture worth $1.9bn to develop alpha-emitting cancer therapies, underscoring the importance of this space to big pharma.
A court order encompassing funding, drug pricing, clinical trials and overall policy implementation aspects is expected to bring about a paradigm shift in the treatment of rare diseases in India. Sarepta, Roche and Sanofi are among the key players that have been part of pricing discussions.
Big pharma executives at ARM’s Cell and Gene Meeting on the Mesa shared some of the practical challenges of bringing one-time treatments to market in hemophilia, cancer and beyond.
Impressed by the data to date on bexicaserin for epileptic encephalopathies syndromes, the Danish drugmaker hopes the drug, through the acquisition of Longboard, will become a cornerstone of its new neuro-rare disease franchise.
While vatiquinone may have missed its primary endpoint in a Phase III trial, PTC is ready to file the drug for the debilitating, life-shortening disorder after showing that it slowed disease progression over 144 weeks.
With more advanced therapies gaining approval, companies at ARM’s Cell and Gene Meeting on the Mesa considered hurdles to making their products more broadly available and developing them for larger indications.
Phase III success positions Scholar Rock’s selective myostatin inhibitor for regulatory filings in early 2025. Analysts see a blockbuster opportunity for additive therapy in spinal muscular atrophy.
Once the deal with Sanofi closes, the rare disease drug will become one of Recordati’s fastest forecast sellers.
CEO Chris Cargill talks to Scrip about Sosei Heptares’ new identity as Nxera, the ups and downs of being big pharma’s go-to small-molecule drug hunter and its move into commercialization in Japan.
Avidity hopes to complete enrollment in mid-2025 for an ongoing Phase III trial of its antibody-oligonucleotide conjugate in myotonic dystrophy type 1, which has no approved drug therapy.
An upfront payment of $110m from BMS has come just in time for Prime Medicine, but its real test will be the first Phase I data, expected early next year.