Novartis Submits CTA for OAV101 Gene Therapy in China, Bringing Hope to SMA Patients

　　【Pharmaceutical Network Product News】According to public information from the Center for Drug Evaluation (CDE) of the National Medical Products Administration, the clinical trial application for OAV101 Injection submitted by Novartis in China has been accepted, as listed in the "Accepted Products" column.

 

It is reported that in April 2018, Novartis announced it would acquire AveXis for $8.7 billion to advance the development of OAV101. In December 2018, Novartis submitted a marketing application for this gene therapy to the FDA, which subsequently received FDA approval for commercialization in 2019. Priced at up to $2.125 million (approximately 11.02 million RMB), it is primarily indicated for the treatment of a rare disease known as spinal muscular atrophy (SMA). This is a severe neuromuscular disorder characterized mainly by progressive, symmetrical weakness and atrophy of the proximal muscles in the limbs and trunk.
 

OAV101 delivers a transgene encoding the survival motor neuron (SMN) protein via an AAV9 viral vector, with the transgene engineered to enhance its capacity to produce the SMN protein. It is designed to enable long-term expression of the SMN protein in cells following a single administration, thereby achieving a "curative" effect. Previously, this product has been granted Breakthrough Therapy designation by the FDA, PRIME designation by the European Union, and Sakigake (innovative drug) designation by Japan.
 

Currently, OAV101 has been approved in over 40 countries, with Q3 sales continuing to grow by 28% to reach $375 million, and it is expected to enter the "billion-dollar club" this year.
 

In China's pharmaceutical market for the treatment of spinal muscular atrophy, the main products include nusinersen sodium injection (Spinraza), which has been labeled a "sky-high-priced drug" after its price per dose was reduced from 700,000 RMB to 550,000 RMB; additionally, the small-molecule oral solution risdiplam (Evrysdi) was also approved in June 2021.
 

Prior to its entry into China, the OAV101 injection had already attracted repeated media attention. Many believe that the prohibitively high price of this gene therapy is misaligned with China's reimbursement policies, which may pose significant challenges to its market entry.
 

Rare diseases are characterized by a wide variety of conditions but limited treatment options, resulting in a substantial unmet medical need for patients. Industry experts note that the advantage of gene therapy lies in its ability to replace or correct defective genes, yielding significant therapeutic efficacy. Furthermore, compared to chemical drug development, diseases caused by single-gene mutations have well-defined pathogenesis and clear therapeutic targets. A single administration can provide long-lasting efficacy, which may also help reduce costs.
 

In recent years, multinational pharmaceutical companies such as Takeda, Sanofi, AstraZeneca, and Pfizer have been intensifying their strategic investments in China's rare disease market. Concurrently, relevant policies continue to be advanced to encourage domestic pharmaceutical enterprises to develop rare disease therapeutics, thereby improving drug accessibility for patients in China. Currently, China's rare disease pharmaceutical industry remains in its nascent stage. Backed by a series of supportive policies, industry consensus holds that rare disease drugs are entering a window of opportunity, yet significant challenges persist. On the one hand, the payment and reimbursement system for high-cost rare disease medications in China remains underdeveloped; on the other hand, corporate responsibility is another critical aspect. With advancements in emerging technologies such as gene therapy and RNA therapy, patients with rare diseases are poised to gain more treatment options. However, these innovative products inevitably face premium pricing during their early commercialization phases. How to enhance patient access to these medications remains an issue that requires continuous exploration.