MFDS rejects Genexine's Efesa application, company plans resubmission

Genexine, a Kosdaq-listed biotech, plans to resubmit its application for approval of long-acting anemia drug Efesa (efepoetin alfa) after regulators rejected the filing over manufacturing and quality-control documentation, delaying the company's efforts to bring the treatment to its home market.

(Credit: Genexine)

The Ministry of Food and Drug Safety (MFDS) rejected the application for Efesa, known developmentally as GX-E4, citing the need for additional supporting data related to chemistry, manufacturing and controls (CMC) and a risk management plan (RMP), the company said in a regulatory filing on Friday.

Genexine shares were down 4 percent to trade at 3,350 won ($2.2) at 3:00 p.m. Friday.

Genexine said the MFDS did not raise any issues related to the drug's safety or efficacy during the review process.

"The reason for the rejection was quality-related supplementary requirements that are sufficiently addressable," the company said in a statement. It added that it plans to work with the MFDS to provide the requested data and seek approval again as soon as possible.

Efesa is a long-acting erythropoiesis-stimulating agent (ESA), a class of drugs that stimulates the production of red blood cells (RBC), and is being developed to treat anemia in patients with chronic kidney disease (CKD).

The filing covered patients with non-dialysis chronic kidney disease (ND-CKD), a form of kidney disease in which patients have not yet started dialysis treatment.

Genexine submitted the application to the MFDS in January 2024 after reporting positive phase 3 clinical trial results in December 2023.

In the trial, Efesa was compared with Roche's Mircera (methoxy polyethylene glycol-epoetin beta), a long-acting treatment for anemia in CKD patients.

The company reported hemoglobin (Hb) response rates of 75.6 percent for Efesa and 69.3 percent for Mircera in ND-CKD patients, meeting the study's non-inferiority criteria. Genexine also said the drug showed a favorable safety profile.

The rejection delays what could become one of the company's first domestically approved new drugs, though Genexine said the setback does not affect the drug's clinical value or broader development plans.

Efesa has already received marketing approvals in Indonesia and Cambodia, while regulatory reviews are ongoing in other countries, Genexine said.

Separately, the company is conducting a global phase 3 trial of Efesa in dialysis-dependent CKD patients. Genexine said those development and regulatory activities are proceeding as planned.

The company did not disclose specific CMC deficiencies identified by the MFDS.