Sanofi Showcases Three Innovative Rare Disease Therapies at CIIE, Offering New Hope for Patients with Fabry Disease, Hemophilia B, and MPS I

(November 6, 2019Med Valley(Reporter Zhang Rongrong)There are approximately 7,000 known rare diseases worldwide, yet only about 6% of them have available treatments. “Hoping to use medication at least once before passing away” is the sole aspiration for many rare disease patients during their brief lives. As a pioneer and leader in the field of rare disease treatment, Sanofi introduced three innovative drugs for rare diseases at the Second China International Import Expo, aiming to help patients in China with Fabry disease, Hemophilia B, and Mucopolysaccharidosis Type I confront health challenges and ignite hope for life.

Pioneering Efforts to Actively Promote Access to Rare Disease Medications

Rare diseases, also known as orphan diseases, refer to conditions with extremely low incidence rates, affecting a small total number of patients, and potentially life-threatening. Globally, the research and development (R&D) of drugs for rare diseases presents a significant scientific challenge. Due to the small patient population and limited market demand, the R&D cycle and costs for these drugs are substantially higher than those for conventional medications. In the vast majority of countries, including China, the availability of clinical treatments falls far short of therapeutic needs, leaving many patients with rare diseases without effective medical options.

Despite substantial investments, a challenging process, and limited returns, Sanofi has continued to press forward. Over the past two decades, leveraging its exceptional R&D capabilities, Sanofi has remained committed to providing innovative therapies for patients with rare diseases in China, including Cerezyme® for Gaucher disease, Myozyme® for Pompe disease, Rilutek® for amyotrophic lateral sclerosis (ALS), and Aubagio® for multiple sclerosis. Meanwhile, Sanofi has actively collaborated with various stakeholders across society to enhance the accessibility of rare disease medications, explore diversified patient assistance models, and create space and opportunities for the rapid development of the rare disease sector in China.

Looking Ahead: Accelerating the Introduction of New Drugs Urgently Needed for Rare Disease Clinical Care

At this year’s China International Import Expo, Sanofi unveiled three new drugs in the field of rare diseases, including Fabrazyme® for the treatment of Fabry disease, Alprolix® for the treatment of hemophilia B, and Aldurazyme® for the treatment of mucopolysaccharidosis type I.

Fabry disease is a lysosomal storage disorder. Patients often experience burning pain, which can last from a few minutes to several days and may recur. In severe cases, the pain disrupts normal daily life and causes serious damage to organs such as the kidneys, heart, brain, and nerves. Without effective treatment, the condition can be life-threatening. Fabrazyme® was the first prescription drug approved by the U.S. Food and Drug Administration (FDA) in 2003 for the treatment of Fabry disease in the United States.

The lack of therapeutic drugs has led to a widespread phenomenon of disability among rare disease patients in China, with the majority forced to spend their lives reliant on wheelchairs. For instance, if hemophilia patients receive prophylactic treatment from childhood, the rate of disability can be significantly reduced. Alprolix®, another product showcased by Sanofi at the China International Import Expo (CIIE), is poised to fill the treatment gap in China for long-acting recombinant clotting factors for Hemophilia B. Approved by the FDA in 2014 as the first therapy for Hemophilia B, Alprolix® is used to prevent or reduce bleeding episodes and is also the world’s first long-acting recombinant clotting factor therapy.

Mucopolysaccharidosis type I (MPS I) is a rare genetic disorder of mucopolysaccharide metabolism caused by lysosomal dysfunction, which can lead to life-threatening complications such as obstructive airway disease, respiratory infections, or cardiac involvement. Aldurazyme® was approved by the FDA in 2003 as the first drug for the treatment of MPS I.

It is understood that there are currently no specific therapies available in China for Fabry disease and Mucopolysaccharidosis Type I, leaving an urgent need to address the clinical treatment requirements of patients. Yu Lei, Head of Rare Diseases at Sanofi China’s Specialty Care Business Unit, stated, “The China International Import Expo (CIIE) is not only a platform for showcasing medical innovations but also an important window for popularizing disease knowledge among the public and promoting scientific treatment concepts. We hope to leverage this CIIE to raise awareness about rare diseases. In the future, Sanofi will continue to deepen its commitment to the rare disease field, leading innovation in research, development, diagnosis, and treatment. We will continue to introduce more groundbreaking global product portfolios and solutions to meet the growing healthcare and health needs of Chinese patients, ensuring that individuals with rare diseases in China receive better treatment and lead lives with dignity.”

(Figure: Yu Lei, Head of Rare Diseases at Sanofi China’s Specialty Care Business Unit)

Moving Forward Together to Realize the “Chinese Dream” for Patients with Rare Diseases

In recent years, the Chinese government has increasingly prioritized the rare disease community. The State Council and the former China Food and Drug Administration (CFDA) have successively issued regulations and implementation measures for special approval, dedicated channels, priority review, and accelerated market launch of drugs for rare diseases. In February this year, at an executive meeting of the State Council convened by Premier Li Keqiang, it was emphasized that medication access for over 20 million rare disease patients must be ensured, and a decision was made to reduce value-added tax (VAT) on 21 rare disease drugs.

“The ‘Chinese Dream’ for patients with rare diseases is simply to have access to affordable medications,” pointed out Professor Li Dingguo, Chairman of the Shanghai Rare Disease Prevention and Treatment Foundation. “In China, only 55 drugs with clearly registered indications for rare diseases are available, covering 31 of the 121 rare diseases listed in the First Batch of the Rare Disease Catalog. There are still 21 rare diseases for which treatments are available abroad but not in China. Accelerating the introduction of new overseas drugs for rare diseases is the first step in ensuring medication access for patients. The Chinese government has made significant progress in accelerating the market approval of drugs for rare diseases. Only by first ensuring that patients ‘have drugs available’ can we coordinate multi-party efforts to overcome the challenge of ‘affordability,’ making the arrival of better days for rare disease patients no longer a distant dream.”

(Figure: Professor Li Dingguo, Chairman of the Shanghai Rare Disease Prevention and Treatment Foundation)

Zhejiang ProvinceMedicineXie Junming, President-Elect of the Rare Disease Branch, stated, “Addressing rare diseases is a significant social undertaking that requires the collaborative participation of various sectors of society. In recent years, the state has successively introduced multiple healthcare policies benefiting the public, thereby helping an increasing number of patients. Looking ahead, we recommend providing fiscal underwriting for high-cost orphan drugs, establishing a national special fund for rare disease coverage, and exploring multi-party financing models for security. Only through joint efforts and collaboration among all stakeholders can patients with rare diseases truly embrace a new lease on life.”

(Figure: Xie Junming, President-elect of the Rare Disease Branch of Zhejiang Medical Association)