China Fast-Tracks Four Innovative Drugs for Breakthrough Therapy Designation from Haisco, Merck, and Domestic Biotechs

Beijing

China's drug regulator is fast-tracking four innovative medicines from Haisco Pharmaceutical Group, Merck, and two domestic biotechs, signaling Beijing's push to close the gap between lab breakthroughs and patients in need.

The Center for Drug Evaluation, or CDE, under the National Medical Products Administration, has proposed listing four drugs under its Breakthrough Therapy Designation program. The drugs — submitted between September 14 and September 17 — target brain tumors, cold sores, a rare inflammatory skin disease, and breast cancer.

Unlike routine regulatory filings, the breakthrough designation is reserved for medicines that show early promise against serious conditions where few treatment options exist. It grants developers accelerated review and closer engagement with regulators — but it is not a green light for sale. Each drug must still clear full clinical trials and a comprehensive CDE review before reaching the market.

A Brain Tumor With Few Options

Haisco Pharmaceutical Group's HSK42360-Na tablets, submitted on September 17, aim at one of the most aggressive cancers in the central nervous system: high-grade glioma with a BRAF V600 mutation.

These tumors grow fast, infiltrate surrounding tissue, and almost always return. The BRAF V600 mutation — most commonly the V600E variant — drives malignancy by keeping the MAPK signaling pathway permanently switched on. It accounts for roughly 5% to 10% of high-grade gliomas in children and adolescents, and an even higher share in certain pathological subtypes.

Standard chemotherapy and radiation offer limited benefit. HSK42360-Na, a next-generation BRAF "paradox breaker" developed in-house by Haisco, is designed to shut down the mutant BRAF protein without triggering the counterproductive reactivation of the same pathway that has bedeviled earlier drugs. The compound is a sodium salt optimization of HSK42360.

A $10.8 Billion Bet

Merck's MK-7240 injection, submitted on September 16, targets moderate to severe hidradenitis suppurativa — a rare, chronic inflammatory skin condition that causes painful deep nodules, abscesses, and tunneling scars in skin folds.

The drug is a humanized monoclonal antibody that blocks tumor necrosis factor-like ligand 1A, or TL1A, a protein involved in immune regulation and inflammation. It was originally developed by Prometheus Biosciences, which Merck acquired in June 2023 for approximately $10.8 billion in upfront payment — one of the largest biotech buyouts in recent years.

MK-7240 is now being studied across multiple indications, reflecting Merck's broader bet on the TL1A pathway.

One Dose for Cold Sores

Huinuo Bio's HN0037 tablets, also submitted on September 16, take aim at recurrent herpes labialis — the medical term for cold sores caused by the herpes simplex virus.

The condition produces clusters of painful, burning blisters around the mouth. HN0037 is a novel helicase-primase inhibitor that shows strong activity against both HSV-1 and HSV-2. Its long half-life means it could treat an entire episode with a single dose — a potential advantage over current antivirals that require multiple doses over several days.

A Dual-Target Weapon Against Breast Cancer

Sichuan Biokin Pharmaceutical Co., Ltd.'s BL-B01D1 for injection, submitted on September 14, is an EGFR×HER3 bispecific antibody-drug conjugate — a molecule engineered to latch onto two cancer-cell targets at once and deliver a cytotoxic payload directly to the tumor.

The proposed indication is first-line treatment of PD-L1-positive (CPS≥1) locally recurrent, unresectable, or metastatic triple-negative breast cancer, in combination with toripalimab, a PD-1 inhibitor.

BL-B01D1 is already being tested in more than 30 clinical trials across China and the United States, covering over a dozen tumor types — a sign of the ambition behind Biokin's pipeline.

The Road Ahead

Breakthrough therapy designation is a stamp of early clinical promise, not a guarantee of approval. Each of the four drugs must complete later-stage trials and pass the CDE's full review before any can be sold commercially.

Still, the batch of submissions underscores a broader shift in China's pharmaceutical landscape. As regulators refine review pathways and companies pour resources into first-in-class and best-in-class candidates, medicines targeting unmet needs — from rare skin diseases to hard-to-treat brain cancers — are moving through the pipeline faster than ever.

For patients with few options, that pace can't arrive soon enough.