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Home » Topics » Regulatory » Orphan drug

Orphan drug
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High-density lipoprotein particles and red blood cells
Endocrine/metabolic

Repair Biotechnologies’ REP-0003 designated orphan drug for homozygous familial hypercholesterolemia

May 20, 2025
No Comments
Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).
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Cerebellum, brain stem, spinal cord
Neurology/psychiatric

Cure Rare Disease’s CRD-002 awarded orphan drug designation for spinocerebellar ataxia type 3

May 15, 2025
No Comments
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).
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Approved stamp

South Korea approves record number of biosimilars in 2024

May 6, 2025
By Marian (YoonJee) Chu
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.
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Approved stamp

South Korea approves record number of biosimilars in 2024

April 30, 2025
By Marian (YoonJee) Chu
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.
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Illustration of kidney with DNA structures
Nephrology

Purespring’s PS-002 designated orphan drug for IgA nephropathy

April 28, 2025
The EMA has granted European orphan drug designation to Purespring Therapeutics Ltd.’s PS-002 for IgA nephropathy (IgAN).
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Illustration of double helix
Endocrine/metabolic

Innorna’s mRNA therapy granted US orphan drug designation for Wilson disease

April 28, 2025
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.
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Neurology/psychiatric

FDA grants orphan drug designation to BA-102 for Phelan-McDermid syndrome

April 22, 2025
Neuronos Ltd., a subsidiary of Beyond Air Inc., has announced BA-102 has been awarded U.S. orphan drug designation for the treatment of Phelan-McDermid syndrome.
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3D rendering of skin cells and elastin with collagen layer
Dermatologic

Resvita Bio’s RVB-003 designated orphan drug for Netherton syndrome

April 4, 2025
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.
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Tape measure wrapped around scale
Endocrine/metabolic

PL-7737 gets orphan drug status for leptin receptor deficiency

March 26, 2025
Palatin Technologies Inc. has obtained U.S. orphan drug designation for PL-7737 for leptin receptor (LEPR) deficiency, including obesity caused by this condition.
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Coronavirus, lungs

Healios posts more upbeat data on Multistem cell therapy for ARDS

March 18, 2025
By Marian (YoonJee) Chu
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.
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