Transition Bio and Voyager Therapeutics forge strategic collaboration to target ALS, FTD

The partnership aims to develop novel, selective small molecules targeting TDP-43 pathology

FDA grants Orphan Drug Designation for Tinostamustine in malignant glioma

Tinostamustine is an investigational drug with a potential first-in-class mechanism combining bifunctional alkylating activity and pan histone deacetylase (HDAC) inhibition

Biohaven disappointed as rare disease therapy faces FDA setback 'despite promising results'

The FDA setback highlights ongoing tensions between patient needs, regulatory processes, and the use of real-world evidence in evaluating treatments for rare diseases

Valneva’s next-gen Zika vaccine shows strong safety and immune response in phase I trial

VLA1601 is a purified and inactivated vaccine specifically designed to protect against the Zika virus

Zydus receives FDA’s ODD for Desidustat for the treatment of beta-thalassemia

Orphan drug designation (ODD) by the USFDA for Desidustat, provides eligibility for a potential seven-year marketing exclusivity subject to the USFDA approval

FDA approves UCB’s Kygevvi for rare genetic mitochondrial disease TK2 deficiency

Kygevvi combines two pyrimidine nucleosides, doxecitine and doxribtimine, which act by integrating deoxycytidine and deoxythymidine into skeletal muscle mitochondrial DNA

Roche’s Gazyva shows breakthrough efficacy in phase III lupus study

The ALLEGORY study demonstrated statistically significant improvements in key measures, including British Isles Lupus Assessment Group-based Composite Lupus Assessment (BICLA) response

Industry leaders call for a new era of collaboration, innovation, and self-reliance

Building a transparent, data-driven supply chain and self-reliant API ecosystem remains critical to reducing import dependence

Medanta Super Speciality Hospital to set up new hospital in Guwahati

The upcoming state-of-the art 400+ bed facility is set to come up at an approximate cost of Rs 500 crore

FDA rejects Regeneron’s Eylea HD pre-filled syringe application amid manufacturing setbacks