Rare Diseases
The Swedish company is celebrating approval for the first treatment for MCT8 deficiency
The Paris-headquartered group has been reflecting on a solid performance last year.
After coming up blank with its in-house oncology efforts, the German conglomerate is mulling a deal to acquire the US specialist company which has just managed a new drug approval for mirdametinib.
CEO Djordje Filipovic tells Scrip that after talks with "multiple other potential partners,” the firm chose the Japanese group for its expertise and marketing capabilities in rare diseases” to be the licensing partner for tadekinig alfa in the US.
The Danish drugmaker, best known for dermatology, has secured the European rights to Shanghai Junshi Biosciences’ cancer therapy toripalimab, which was the first Chinese PD-1/L1 checkpoint inhibitor to be approved in the US.
On the back of a stellar showing in Phase III, apitegromab is going to be filed in the US and Europe in the coming weeks for spinal muscular atrophy and CEO Jay Backstrom believes it will be a $2bn blockbuster.
The US biotech’s Duchenne muscular dystrophy drug is “undoubtedly the most successful gene therapy launch in all of history,” according to CEO Doug Ingram. “We have barely scratched the surface of the opportunity in front of us.”
Two treatments that target elF2B, a regulator of the integrated stress response that appears to be overactive in the progressive motor neurone disease, have missed their primary and secondary endpoints in a landmark platform trial.
From gene therapies to small molecule drugs, investors seemed to have eschewed the risks of biotech company investment propositions in 2024 but last year’s disappointments provided good lessons going forward.
Taiwan-based PharmaEssentia hopes to add essential thrombocytopenia to the label for its pegylated interferon product, already approved to treat polycythemia vera.
The Swiss-headquartered, NASDAQ-listed group has posted a Phase II win for OCS-05 in acute optic neuritis, which opens up its potential in additional, and much larger, neuro-ophthalmic indications, CEO Riad Sherif tells Scrip.
The Swiss giant’s blockbuster gene therapy is currently only administered intravenously into patients with spinal muscular atrophy under two years of age. Its intrathecal candidate, called OAV-101, has just passed a Phase III test which could expand its use in children and young adults.
UniQure said discussions with the FDA resulted in a path forward for seeking accelerated approval of its gene therapy AMT-130 in Huntington’s disease.
Primary biliary cholangitis or cirrhosis, PBC, is a lesser-known cousin of MASH but a silent killer, nevertheless. Scrip studies data from Citeline’s Pharmaprojects and separately Evaluate Pharma to reveal a promising pipeline and drug revenue forecasts, with APAC firms accounting for half the pipeline of drugs
Novartis will also pay up to $1.9bn for milestones and share US profits on the Phase II RNA-splicing candidate. PTC is discussing potential accelerated approval with the US FDA.
Deal Snapshot: The essential tremor space has not seen innovation in treatment for decades. Acadia believes Saniona's GABAA-α3 positive allosteric modulator could be the answer.
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.