Rare Diseases
The European Medicines Agency is set to adopt opinions on whether marketing approval should be granted to five new products, including three orphans. A decision is also due on a previously-rejected Alzheimer’s drug that was under re-examination.
Several orphan drugs, including SOBI’s emapalumab, Merck KGaA’s pimicotinib and Percigen’s zopapogene imadenovec, are now under review for potential pan-EU marketing authorization by the European Medicines Agency.
A marketing authorization application for another AstraZeneca product – its orphan drug candidate anselamimab – is now also under review in the EU.
The FDA cited various issues with the CAMPSIITE trial in Hunter syndrome and offered multiple paths forward, but the biotech said that could be a challenge given the rarity of the disease.
The company announced positive Phase II results for the JAK1/TYK2 inhibitor in cutaneous sarcoidosis, which has no approved treatments.
The European Medicines Agency is set to adopt opinions on whether marketing approval should be granted to five new products, including therapies for a serious liver disease and an ultra‑rare genetic mitochondrial disorder.
ANI’s Cortrophin Gel is benefiting from significant under-penetration in areas such as nephrology, neurology, rheumatology and pulmonology, the firm told attendees to the recent J.P. Morgan Healthcare Conference.
Biogen’s head of development reflects on transforming company strategy, surviving the Aduhelm crisis and building sustainable growth through disciplined portfolio management.
The company plans to take Aqneursa to regulators in the US, Europe and elsewhere for ataxia-telangiectasia (A-T), which currently lacks any approved therapy.
Zydus is to gain from a world-first nod for a Menkes disease drug in the US amid speculation on a deal to acquire Ardelyx. Scrip examines how Zycubo approval, Agenus’s oncology BOT/BAL combination progress and a Formycon partnership help the Indian major
The Niemann-Pick disease type C drug is off to a good start in the US but Europe may be even more lucrative, CEO Neil McFarlane tells attendees at the J.P. Morgan meeting.
Amphastar is looking to a “strategically important addition to our growing proprietary peptide portfolio,” after striking an exclusive licensing agreement with China-based Nanjing Hanxin Pharmaceutical Technology.