Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).

The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia type 3 (SCA3).

South Korea’s Ministry of Food and Drug Safety approved 18 biosimilar products in 2024, making it a record year for domestic biosimilar approvals since the agency’s first nod of Celltrion Inc.’s Remsima, a reference product of Remicade (infliximab), in 2012.

South Korea’s Ministry of Food and Drug Safety approved 18 biosimilar products in 2024, making it a record year for domestic biosimilar approvals since the agency’s first nod of Celltrion Inc.’s Remsima, a reference product of Remicade (infliximab), in 2012.

The EMA has granted European orphan drug designation to Purespring Therapeutics Ltd.’s PS-002 for IgA nephropathy (IgAN).

Innorna Co. Ltd.’s investigational mRNA therapy, IN-013, has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of Wilson disease.

Resvita Bio Inc.’s RVB-003 has been awarded orphan drug designation by the FDA for the skin disorder Netherton syndrome. RVB-003 was previously granted rare pediatric disease designation.

Palatin Technologies Inc. has obtained U.S. orphan drug designation for PL-7737 for leptin receptor (LEPR) deficiency, including obesity caused by this condition.

Latest findings on Healios K.K.’s stem cell therapy to treat acute respiratory distress syndrome (ARDS), coined Multistem (invimestrocel; HLCM-051), found the regenerative medicine effective in reducing the number of patient days on ventilator treatment, as well as mortality benefits.