HOUSTON, Texas — IPS HEART, Inc., a biopharmaceutical company pioneering induced pluripotent stem cell (iPSC)-derived cell replacement therapies, announced that the FDA has granted Rare Pediatric Drug Designation for ISX9-CPC, for the treatment of cardiomyopathies associated with dystrophinopathies. Redefining the Therapeutic Paradigm: Generating New Human Muscle Duchenne muscular dystrophy...
Latest News
PARIS, France and BOSTON, Mass. — Ipsen (Euronext: IPN; ADR: IPSEY) and Skyhawk Therapeutics today announced the signing of an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases. The agreement includes an option pursuant to which Ipsen would acquire exclusive license...
Ipsen announced it has completed the acquisition of Albireo Pharma, Inc., a leading innovator in bile-acid modulators to treat rare liver conditions.
PARIS – Ipsen (Euronext: IPN; ADR: IPSEY) today announced that its New Drug Application (NDA) for palovarotene, an oral, investigational, selective RARγ agonist for the prevention of heterotopic ossification (new bone formation) as a potential treatment option for people living with the progressive disabling and ultra-rare genetic disorder fibrodysplasia ossificans progressiva...
PARIS, FRANCE, 19 December 2025 – Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the pivotal Phase II FALKON trial did not meet its primary endpoint of reducing new heterotopic ossification (HO) in adults and children living with fibrodysplasia ossificans progressiva (FOP) vs. placebo, as a result the study will be...
PARIS, France – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Phase III BOLD trial evaluating Bylvay versus placebo for patients living with biliary atresia (BA) who have already undergone a Kasai hepatoportoenterostomy (HPE) did not meet the primary endpoint of improvement in native liver survival vs placebo. Current topline...
Seven abstracts, including three late-breaking presentations, will be shared on new IQIRVO® (elafibranor) and Bylvay®/Kayfanda® (odevixibat) data, reinforcing the strong clinical value across Ipsen’s rare liver disease portfolio where unmet need is high and treatments are few In two late-breaking presentations, IQIRVO’s effect on fatigue and its relationship with pruritus in patients...
Paris, France – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted regulatory approval for Bylvay® (odevixibat) for the treatment of pruritus associated with progressive familial intrahepatic cholestasis (PFIC). PFIC is a group of rare genetic disorders in which bile acid accumulates in the...
PARIS, FRANCE – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion recommending the conditional marketing authorization of Ojemda® (tovorafenib) as monotherapy for the treatment of patients 6 months of age and...
PARIS, France – Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Phase IIIb ELSPIRE trial evaluating IQIRVO® (elafibranor) in patients with PBC with an Alkaline Phosphatase (ALP) 1-1.67 times the upper limit of normal (x ULN) met the primary endpoint with statistical significance, showing an ALP normalization rate of 85%...