Rare Diseases
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.
IntraBio and Zevra will go head-to-head in the treatment of Niemann-Pick disease type C, with US FDA approvals of Aqneursa and Miplyffa, respectively, less than a week apart.
Rocatinlimab met its Phase III endpoints with results that appear uncompetitive with AD market leader Dupixent, while Uplizna may offer a compelling twice-yearly gMG treatment option.
The exon 53 skipper WVE-N531 looks highly competitive with approved therapies, but a biomarker hit might not correlate with functional improvement.
Miplyffa (arimoclomol) was approved for Niemann-Pick disease type C when administered with another high-priced drug used off label as standard of care for about 80% of NPC patients.
The EDG-7500 results have seen the company’s share price rocket, thanks to a potential advantage over existing treatments for the rare heart condition hypertrophic cardiomyopathy.
While it waits to see if Fasenra succeeds in a Phase III trial for chronic obstructive pulmonary disease, the UK major has got over the regulatory line in the much smaller but underserved space of eosinophilic granulomatosis with polyangiitis.
The Danish company released pivotal data for TransCon CNP in children with achondroplasia that appears competitive with BioMarin’s big growth driver.
The Duchenne drug Agamree has started so well in Germany and Austria that the Swiss company is expanding its self-marketing strategy in the top five European markets to include the Nordics, Portugal and Ireland.
The Roivant-owned company announced positive Phase II data for batoclimab in Graves’ disease and plans to advance next-generation IMVT-1402 into Phase III.
A Phase III trial with oral deucrictibant is underway for treatment of acute hereditary angioedema attacks, while the Swiss firm will begin a Phase III trial in prophylaxis later this year.