GSK’s experimental lung cancer drug gains FDA orphan drug status

GSK holds exclusive global rights (excluding mainland China, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to advance its development and commercialization

Praxis Precision hits milestone in fight against rare paediatric seizures

The EMBOLD study evaluated relutrigine for patients with SCN2A and SCN8A developmental and epileptic encephalopathies

Bayer advances fight against rare kidney disease with new experimental drug

Alport Syndrome is caused by genetic mutations affecting type IV collagen in the kidneys, ears, and eyes

Merck KGaA’s Cladribine capsules gain FDA fast track for rare neuromuscular disorder

FDA grants accelerated approval to sevabertinib for non-squamous non-small cell lung cancer

HER2-mutant NSCLC typically has a poor prognosis and limited treatment options

Cardiol Therapeutics wins US patent protection for heart-disease drug platform

The patent will lock in broad protection for the company’s lead therapy CardiolRx and its novel subcutaneous candidate CRD-38

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

Sanofi’s Efdoralprin Alfa shows superiority in phase 2 Alpha-1 antitrypsin deficiency study

Efdoralprin Alfa was well tolerated, with a safety profile comparable to plasma-derived therapy

Ipsen to acquire ImCheck Therapeutics to strengthen oncology pipeline

The acquisition centers on ImCheck’s lead Phase I/II program, ICT01, a first-in-class monoclonal antibody targeting BTN3A

Glenmark signs cancer drug licensing deal with Hengrui Pharma for $18 million upfront payment

Hengrui is eligible to receive regulatory and commercial milestone payments of up to US$1.093 billion