Third Failure! Rare Disease Drug Effort Wiped Out

May 26, 2026 11:27 CST Updated 11:27

Gowell Pharma

Cosmetics and Pharmaceutical R&D Developers

Qilu Pharmaceutical

Specialty Formulations and Active Pharmaceutical Ingredients (API) Developer

Ionis Pharmaceuticals

RNA Drug Developer

Biogen

Neuroscience Drug Developer

On May 25, the latest drug notification delivery information (officially rejected/voluntarily withdrawn by the company) from China's National Medical Products Administration (NMPA) showed that:

The marketing authorization application for Nusinersen Sodium Injection by Chengdu Gowell Biopharmaceutical Co., Ltd. (Acceptance No.: CYHS2501093) is listed.

Information from the Center for Drug Evaluation (CDE) indicates that the NDA application was accepted on March 18, 2025 (Registration Category: Class 4 Generic).

Notably, prior to Chengdu Gowell, two pharmaceutical companies had already failed. To date, all companies that have filed applications for this product have been unsuccessful.

In April this year, Qilu Pharmaceutical Co., Ltd.'s generic nusinersen sodium injectionThe marketing authorization application (Acceptance No.: CYHS2500029) appears in the drug notification document.

In November 2025, Chongqing Pharmed's generic nusinersen injectionThe Marketing Authorization Application (Acceptance No.: CYHS2402891) appears in the drug notification document.

Spinraza (Nusinersen/NuoNusinersen sodium) is a modified antisense oligonucleotide (ASO) that binds to the splicing site of exon 7 in SMN2, reducing the likelihood of truncation and increasing the production of full-length SMN protein, thereby treating the disease.

Originally co-developed by Cold Spring Harbor Laboratory and Ionis Pharmaceuticals, in 2015BoHealthSecure global development, manufacturing, and commercialization rights.

December 2016, approved by the FDA for marketing, brand name:Spinraza，to become the first drug for the treatment of SMA in both children and adults.

In February 2019, the NMPA approved via priority review.`Promise`Nusinersen Sodium Injectionsolution launched,For the treatment of 5q spinal muscular atrophy (SMA).

NusinersenNusinersen sodium is renowned for its exorbitant treatment costs, priced at $125,000 per vial following its U.S. launch, totaling870,000 RMB. In China, the price per injection is 700,000 RMB, requiring repeated annual injections, and it is a fully self-pay medication., however, it was included in the national medical insurance list in 2022, with the cost per dose reduced to 30,000 yuan.

Reference: Drug News Commentary

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