FDA approves Merck’s WINREVAIR for treatment of pulmonary arterial hypertension

WINREVAIR is a breakthrough biologic for this rare, progressive disease

USFDA grants ODD to Cevidoplenib for immune thrombocytopenia

Oscotec has successfully completed phase 2 study in patients with chronic ITP last year

Celanese and Secarna Pharmaceuticals collaborate for RNA research for antisense therapies

AstraZeneca to acquire Amolyt Pharma

Acquisition to further expand the Alexion, AstraZeneca Rare Disease pipeline beyond complement inhibition

Moderna posts Q4 2023 revenue at US$ 2.8 billion

Net product sales in the fourth quarter of 2023 include the recognition of $0.6 billion from deferred revenue, related to Gavi, the Vaccine Alliance

ImmunoBrain Checkpoint appoints Dr. Sanjay Keswani as CEO

He is a former Neurology faculty member at The Johns Hopkins Hospital and an elected Fellow of the Royal College of Physicians, United Kingdom

EMA grants ODD to GC Biopharma's treatment for Sanfilippo Syndrome

GC1130A has previously achieved notable milestones by securing both RPDD and ODD from the U.S. FDA in January 2023

European Commission grants ODD to NS-229 for treatment of Eosinophilic Granulomatosis

The orphan drug designation by the EC is issued for drugs which are intended to treat diseases that affect fewer than five in 10,000 people in the European Union

Thermo Fisher receives AIFA’s GMP approval for manufacturing facility of RNA-based products in Italy

GMP certification helps enable biotechnology customers to accelerate accessibility of new RNA-based therapies for patients

Merck to acquire Caraway Therapeutics

Acquisition underscores Merck’s ongoing commitment to developing treatments for neurodegenerative diseases