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BioWorld - Friday, June 20, 2025
Home » Topics » Regulatory » Orphan drug

Orphan drug
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3d rendering of bispecific antibodies
Cardiovascular

Diagonal Therapeutics’ DIAG-723 designated orphan drug for hereditary hemorrhagic telangiectasia

June 19, 2025
No Comments
Diagonal Therapeutics Inc.’s DIAG-723 has been awarded orphan drug designation by the FDA for the treatment of hereditary hemorrhagic telangiectasia (HHT). Additionally, the EMA has provided a positive opinion for orphan drug designation, confirming that DIAG-723 meets the criteria for designation as an orphan drug in the E.U.
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Illustration of cancer tumor on pancreas
Immuno-oncology

Medicovestor’s Adobind MC-001 awarded US orphan drug designation

June 18, 2025
No Comments
Medicovestor Inc.’s Adobind MC-001 has been awarded orphan drug designation by the FDA for the treatment of pancreatic cancer. Adobind MC-001 is a chemoimmunotherapy antibody-drug conjugate that integrates proprietary enhancements to antibody structure, payload delivery and tumor engagement.
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Neuronata-R

Corestemchemon preps US BLA filing for stem cell ALS therapy

June 10, 2025
By Marian (YoonJee) Chu
No Comments
Corestemchemon Inc. is planning to file a BLA for Neuronata-R (lenzumestrocel) by the end of 2025 to gain accelerated approval from the U.S. FDA, company officials confirmed to BioWorld during a June 2 interview. Neuronata-R is an autologous bone marrow-derived mesenchymal stem cell therapy that first gained approval in South Korea in 2014 to delay the progression of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder also known as Lou Gehrig’s disease.
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Neuronata-R

Corestemchemon preps US BLA filing for stem cell ALS therapy

June 9, 2025
By Marian (YoonJee) Chu
No Comments
Corestemchemon Inc. is planning to file a BLA for Neuronata-R (lenzumestrocel) by the end of 2025 to gain accelerated approval from the U.S. FDA, company officials confirmed to BioWorld during a June 2 interview. Neuronata-R is an autologous bone marrow-derived mesenchymal stem cell therapy that first gained approval in South Korea in 2014 to delay the progression of amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disorder also known as Lou Gehrig’s disease.
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Cancer

EMA’s COMP recommends orphan drug designation for Hemispherian’s GLIX-1 for glioma

June 6, 2025
No Comments
The EMA’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending European orphan drug designation for Hemispherian AS’s GLIX-1 for the treatment of glioma.
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Mesothelioma
Immuno-oncology

Oncovita’s Mvdeltac designated orphan drug for pleural mesothelioma

June 5, 2025
No Comments
Oncovita SAS’s lead oncolytic virus candidate, Mvdeltac, has been awarded orphan drug designation by the FDA.
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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.
Read More
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