Numerous K-Bio drugs secure FDA ODD

11 in the first half of 2024·10 in the second half of 2024 receive ODD from the FDA

Companies target various diseases, including solid cancers·pulmonary hypertension·Wilson's disease·Lafora disease

Korean pharmaceutical and biotech companies seek to enter the market for rare diseases through the orphan drug designation.

 

Th rare disease field has a small pool of patients, but companies can obtain an exclusive status by acquiring innovative new drugs when they succeed and create high-added value.

 

New drug candidates from various companies, including Hanmi Pharm, GC Biopharma, GI Innovation, and Rznomics, successfully obtained the U.S.

 

Food and Drug Administration (FDA)'s Orphan Drug Designation (ODD).

 

According to industry sources on December 16, twenty-one new drug candidates under development by Korean pharmaceutical and biotech companies received ODD from the FDA.

 

ODD provides companies developing treatments for patients with rare and intractable diseases, which have a disease prevalence of under 100,000 population, benefits such as expedited review, tax reductions, and exclusive status for new drugs.

 

Pharmaceutical companies, Boryung·GC Biopharma, have secured FDA's ODD in the first half of the year In the first half of this year, new drug candidates by Boryung, Rznomics, NeoImmuneTech, SPARK Biopharma, SN BioScience, Oscotec, Ingenium Therapeutics, Dr.

 

Noah Biotech, Hanmi Pharm, GC Biopharma, and GI Innovation have successfully obtained ODD.

 

First half-year 2024 report: new drug candidates by Boryung, Rznomics, NeoImmuneTech, SPARK Biopharma, SN BioScience, Oscotec, Ingenium Therapeutics, Dr.

 

Noah Biotech, Hanmi Pharm, GC Biopharma, and GI Innovation have successfully obtained ODD.

Boryung's BR-101801, a novel drug candidate to treat blood cancer, was the first to receive FDA ODD this year.

 

Boryung is investigating BR-101801's potential in various blood cancers, including peripheral T-cell lymphoma and mycosis fungoides.

 

In January, it received the approval for the treatment of angioimmunoblastic T-cell lymphoma.

 

BR101801 is the first-in-class drug candidate to inhibit phosphoinositide 3-kinase (PI3K)γ/ δ and DNA-dependent protein kinase (DNA-PK).

 

It can effectively induce cell death through triple target inhibition and suppress a cancer protein c-Myc.

 

NeoImmuneTech's NT-I7 received an ODD in the treatment of pancreatic cancer.

 

NT-I7 is a novel drug candidate that targets interleukin (IL)-7, which regulates T-cell development and function.

 

It has been investigated for various indications.

 

Besides the current ODD for pancreatic cancer, NT-I7 received ODDs in the treatment of CD4 lymphocytopenia (2019), multifocal leukoencephalopathy (2020), and glioblastoma (2023).

 

A Fabry disease treatment, LA-GLA, developed jointly by Hanmi Pharm and GC Biopharma, successfully received an ODD in the United States.

 

LA-GLA is formulated for once-per-month subcutaneous administration.

 

Fabry disease is a type of lysosomal storage disorder (LSD) resulting from a genetic deficiency in a particular enzyme, leading to metabolic alterations.

 

In June, GI Innovation's GI-102, a candidate immunotherapy for cancer, received FDA ODD.

 

The company is developing GI-102, which acts on CD80 and interleukin (IL)-2.

 

IL-2 is involved in immune cell proliferation and activation, and CD80 blocks CTLA-4, a receptor preventing immune cells from attacking cancer cells.

 

In the second half of this year, 11 new drug candidates received FDA's ODD In the second half of this year, eleven new drug candidates from Korean pharmaceutical and biotech companies received ODD in the United States.

 

Second half-year 2024 report: new drug candidates by K-Bio companies, including Zymedi, Connext, iLeadBMS, HysensBio, and Rezolute, successfully received the FDA

Zymedi successfully received the FDA's ODD for its antibody-drug ZMA001 in July.

 

ZMA001 targets intractable disease, pulmonary hypertension.

 

Pulmonary hypertension is a disease characterized by narrowing of the blood vessels in the lungs, leading to increased pulmonary blood pressure and ultimately causing heart failure.

 

In South Korea, approximately half of the patients with pulmonary hypertension die within five years of diagnosis.

 

While treatments such as phosphodiesterase type 5 (PDE5) inhibitors and calcium channel blockers (CCBs) have been used, they only provide symptom management.

 

More new drugs are needed.

 

ZMA001, a human monoclonal antibody, prevents inflammatory macrophages from infiltrating the lungs, fundamentally inhibiting pulmonary hypertension symptoms from the initial stage.

 

In preclinical animal model studies, ZMA001 demonstrated superior efficacy to existing drugs.

 

In August, Connext's acute graft-versus-host disease (GVHD) treatment, 'CNT101,' was added to the FDA's ODD list.

 

CNT101 is a recombinant protein that targets the TLR5 receptor expressed on epithelial and immune cells and is being developed as a treatment for acute radiation syndrome caused by radiation exposure.

 

Connext explains that CNT101 minimizes gastrointestinal tissue damage resulting from radiation toxicity during hematopoietic stem cell transplantation, thereby preventing the onset of GVHD.

 

In October, a Target Protein Degrader (TPD) molecular glue from iLeadBMS, Ildong Pharmaceutical's subsidiary specializing in new drug development, was designated as the FDA's ODD.

 

iLeadBMS is developing a molecular glue that targets cyclin-dependent kinase 12 (CDK12), a protein that controls the expression of cancer-related genes.

 

A complex formation between CDK12 and Cyclin-K is known to influence the growth and metastasis of cancer cells in refractory cancer.

 

Earlier this year, iLeadBMS presented preclinical results at the ESMO Targeted Anticancer Therapies Congress 2024 (ESMO TAT 2024), demonstrating that its molecular glue effectively inhibits CDK12 activity and induces degradation of Cyclin-K, thereby suppressing the growth of HER2-negative gastric cancer cells.

 

This success led to the designation of the drug as an orphan drug for gastric cancer.

 

In December, HysensBio's treatment for amelogenesis imperfecta received ODD in the United States.

 

Amelogenesis imperfecta is a rare genetic disorder that impairs the formation of enamel on teeth.

 

The disease causes symptoms such as tooth sensitivity, erosion, and fractures, and currently, no effective treatment options are available.

 

Rezolute, Handok’s US subsidiary, received the FDA ODD for its 'RZ358,' a treatment for tumor-mediated hyperinsulinism.

 

VasThera's pulmonary arterial hypertension therapy 'VTB-10' also received the FDA's ODD this month.