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New Promise, Looming Competition For DMD In China

70,000 Patients, No Approved Drugs

 
• By Brian Yang and Kosh Naran

With several new drugs progressing for the estimated 70,000 patients in China with DMD, a recent setback for Nippon Shinyaku's contender and progress for Chinese gene therapy developers are adding both uncertainty and likely competition in an untapped market with pressing medical need.

Muscular dystrophy wheelchair user
DMD patients in China sees promise but also uncertainties • Source: Shutterstock

In a recent discussion forum on China's WeChat social media platform, a group of patients whose sons are suffering from Duchenne muscular dystrophy (DMD) had good news to share - neurologists at one of the nation’s top-ranked medical facilities, the Beijing Union Hospital, could finally prescribe PTC Therapeutics, Inc.'s Emflaza (deflazacort).

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More from China

Transient FY26 For Syngene But Momentum In China +1 Projects

 
• By Anju Ghangurde

As biopharma derisks its business with China ‘rebalancing’ strategies, Syngene could convert majority of such pilots into full program contracts amid a challenging FY2025. The CRDMO also expects business discussions pertaining to a newly acquired US biologics site to mature into commercial opportunities.

Zai’s R&D Head Touts ‘Beauty’ Of Novel Internal Assets

 
• By Dexter Jie Yan

Two potentially first-in-class molecules signify Zai Lab’s renewed drive for the in-house discovery of drugs that it requires “to be innovative, differentiated and to have the potential to make a big difference for patients,” its global R&D head tells Scrip.

CSPC’s Global-First Monospecific ADC Promising In Advanced NSCLC

 
• By Xu Hu

CSPC’s SYS6010, the first monospecific EGFR-targeting antibody-drug conjugate in the clinic for advanced NSCLC, has shown Phase I promise, but could face close competition with another China-originated bispecific.

Akeso Explains Ivonescimab Prelim OS Data After Investors’ Negative Reactions

 
• By Dexter Jie Yan

Akeso explains the circumstances behind the release of preliminary overall survival data from a head-to-head Phase III trial with ivonescimab in lung cancer, which some investors viewed as disappointing.

More from Focus On Asia

NIH Funding Cut Left Less Money On R&D Table, Tariffs Would Add To Woes: Aragen’s CEO

 
• By Vibha Ravi

Funding cuts at the National Institutes of Health, big pharma’s US onshoring moves, GCC partnerships and talks with the Indian government to solve intellectual property challenges are discussed in an interview with Aragen’s CEO, who is also keenly watching the Trump administration’s moves on pharma tariffs

Akeso Explains Ivonescimab Prelim OS Data After Investors’ Negative Reactions

 
• By Dexter Jie Yan

Akeso explains the circumstances behind the release of preliminary overall survival data from a head-to-head Phase III trial with ivonescimab in lung cancer, which some investors viewed as disappointing.

Novotech CEO On Flexible Trial Solutions Amid Tariff, Geopolitical Tensions

 
• By Anju Ghangurde

Novotech's CEO talks about rising interest for trials in regions with “regulatory agility” and “strategic insulation” from geopolitical complexity, especially among emerging and mid-sized biotech sponsors. Operational "reassessment” among some sponsors on exposure to China-based CROs/CRDMOs is another area he discussed.