• Sign In
  • Sign Out
  • My Account
Subscribe
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
  • Special reports
Clarivate
  • Data Snapshots
  • BioWorld
  • BioWorld MedTech
  • Infographics: Dynamic digital data analysis
  • Index insights
  • Special reports
  • Infographics: Dynamic digital data analysis
  • Trump administration impacts
  • Biopharma M&A scorecard
  • BioWorld 2024 review
  • BioWorld MedTech 2024 review
  • BioWorld Science 2024 review
  • Women's health
  • China's GLP-1 landscape
  • PFA re-energizes afib market
  • China CAR T
  • Alzheimer's disease
  • Coronavirus
  • More reports can be found here

BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe
Home » Topics » Regulatory » Orphan drug

Orphan drug
Orphan drug RSS Feed RSS

Multiple myeloma illustration
Immuno-oncology

Brightpath’s BP-2202 designated orphan drug for multiple myeloma

July 4, 2025
No Comments
Brightpath Biotherapeutics Co. Ltd.’s iPS cell-derived BCMA CAR-natural killer T cell therapy candidate has been awarded orphan drug designation by the FDA for the treatment of multiple myeloma.
Read More
DNA and cancer cells
Cancer

Hemispherian’s GLIX-1 awarded US orphan drug designation for malignant glioma

July 1, 2025
No Comments
GLIX-1 is a first-in-class small-molecule therapeutic targeting DNA repair vulnerabilities in cancer cells.
Read More
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.
Read More
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.
Read More
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.
Read More
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.
Read More
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.
Read More
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.
Read More
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).
Read More
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).
Read More
Previous 1 2 3 4 5 6 7 8 9 … 12 13 Next

Popular Stories

  • Today's news in brief

    BioWorld
    BioWorld briefs for July 3, 2025.
  • Today's news in brief

    BioWorld MedTech
    BioWorld MedTech briefs for July 3, 2025.
  • Illustration of the β2-adrenergic receptor

    Exercise-mimicking compound offers alternative to GLP-1 therapies

    BioWorld
    An experimental drug for treating diabetes and obesity has been shown to lower blood sugar levels and increase fat burning. It is a β2-adrenergic receptor (β2AR)...
  • News in brief

    BioWorld Asia
    BioWorld Asia briefs for July 1, 2025
  • Robert Paris, vice president, Moderna

    Moderna expands mRNA tech to latent viruses, cancer, rare disease

    BioWorld
    Post pandemic, Moderna Inc. is broadening the applications of mRNA technology to cancer, rare diseases, latent viruses and respiratory viruses, “taking advantage”...
  • BioWorld
    • Today's news
    • Analysis and data insight
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld MedTech
    • Today's news
    • Clinical
    • Data Snapshots
    • Deals and M&A
    • Financings
    • Newco news
    • Opinion
    • Regulatory
    • Science
  • BioWorld Asia
    • Today's news
    • Analysis and data insight
    • Australia
    • China
    • Clinical
    • Deals and M&A
    • Financings
    • Newco news
    • Regulatory
    • Science
  • BioWorld Science
    • Today's news
    • Biomarkers
    • Cancer
    • Conferences
    • Endocrine/Metabolic
    • Immune
    • Infection
    • Neurology/Psychiatric
    • Patents
  • More
    • About
    • Advertise with BioWorld
    • Archives
    • Article reprints and permissions
    • Contact us
    • Cookie policy
    • Copyright notice
    • Data methodology
    • Infographics: Dynamic digital data analysis
    • Index insights
    • Podcasts
    • Privacy policy
    • Share your news with BioWorld
    • Staff
    • Terms of use
    • Topic alerts
Follow Us

Copyright ©2025. All Rights Reserved. Design, CMS, Hosting & Web Development :: ePublishing

BioWorld. Link to homepage.

Clarivate
  • BioWorld
  • BioWorld MedTech
  • BioWorld Asia
  • BioWorld Science
  • Data Snapshots
    • BioWorld
    • BioWorld MedTech
    • Infographics: Dynamic digital data analysis
    • Index insights
  • Special reports
    • Infographics: Dynamic digital data analysis
    • Trump administration impacts
    • Biopharma M&A scorecard
    • BioWorld 2024 review
    • BioWorld MedTech 2024 review
    • BioWorld Science 2024 review
    • Women's health
    • China's GLP-1 landscape
    • PFA re-energizes afib market
    • China CAR T
    • Alzheimer's disease
    • Coronavirus
    • More reports can be found here

BioWorld. Link to homepage.

  • Sign In
  • Sign Out
  • My Account
Subscribe