FDA Approved Five Gene Therapies in 2023 Targeting Rare Diseases Including SCD and DMD

　　【Pharmaceutical Network Industry Dynamics】Not long after the start of 2024, gene therapy developer Voyager Therapeutics and a subsidiary of Novartis announced a "partnership." The two parties will collaborate to advance clinical-stage gene therapy candidates for Huntington's disease (HD) and spinal muscular atrophy (SMA).
 

Behind the strong collaboration lies the booming growth potential of the gene therapy market. According to research by Allied Market Research and Global Market Research, the global gene editing market size is expected to reach $36.061 billion by 2030, with a compound annual growth rate (CAGR) of 22.3%.
 

CBER Shows That in 2023, CBER Approved 5 Gene Therapies; In 2022, the Division Approved 4 Gene Therapies. The 5 Newly Approved Therapies in 2023 Cover Various Rare Disease Treatment Areas, Including Sickle Cell Anemia (SCD), Duchenne Muscular Dystrophy, and Others.
 

Among them, one is the Casgevy therapy developed based on CRISPR gene editing technology, which is considered a milestone breakthrough in the field of gene therapy.
 

On December 9, 2023, the U.S. FDA announced the approval of a CRISPR/Cas9 gene-editing therapy marketed under the brand name Casgevy for the treatment of patients aged 12 years and older with sickle cell disease (SCD), priced at $2.2 million, equivalent to over 15 million Chinese yuan. Previously, on November 17, 2023, the UK Medicines and Healthcare products Regulatory Agency had approved the therapy for marketing.
 

Data show that Sickle Cell Disease (SCD) is a hereditary blood disorder caused by a mutation in the gene encoding the beta-globin chain of hemoglobin, leading to abnormal hemoglobin function. Clinical manifestations include chronic hemolytic anemia, susceptibility to infections, and recurrent pain crises causing chronic local ischemia, which results in organ and tissue damage. The main treatments are blood transfusions and medication. Patients still face significant unmet clinical needs. On May 11, 2018, the National Health Commission and four other departments jointly formulated the first batch of the rare disease directory, which included sickle cell anemia.
 

Duchenne Muscular Dystrophy (DMD) is also a rare disease. It is a monogenic disorder caused by functional defects in the human DMD gene and is particularly common among progressive muscular dystrophies, with a high incidence rate. Reportedly, without any medication or rehabilitation training, muscle weakness and atrophy in DMD patients will continuously progress, leading to spinal deformities and cardio-pulmonary failure, thereby endangering the patient’s life.
 

On June 22, 2023, Sarepta Therapeutics announced that its gene therapy Elevidys (delandistrogene moxeparvovec, SRP-9001), jointly developed with Roche, received accelerated approval from the U.S. FDA for market launch. This marks the world’s first one-time gene therapy for Duchenne Muscular Dystrophy (DMD), applicable to non-ambulatory pediatric DMD patients aged 4-5 who carry mutations in the dystrophin gene. Sarepta stated that the initial pricing for Elevidys is $3.2 million.
 

According to a report by the industry media Fierce Biotech in July 2023, the director of the FDA's Center for Biologics Evaluation and Research (CBER) stated that gene therapy holds tremendous potential, but its slow progress may be due to obstacles in the regulatory process.
 

To expedite the approval of gene therapy products, the FDA's Center for Biologics Evaluation and Research in the United States has developed a "four-point plan" to accelerate the approval of gene therapies. This includes enhancing production efficiency, confirming expedited regulatory pathways, collaborating with other regulatory agencies, launching the second generation of "Operation Warp Speed" (aimed at advancing the development of drugs for rare diseases), and recommending the simplification of viral vector review procedures. Industry insiders predict that the "four-point plan" is expected to speed up the approval of gene therapies, with an anticipated increase in the number of gene therapy drugs approved by the FDA in the future.
 

Disclaimer: Under no circumstances shall the information or opinions expressed in this article constitute investment advice to any person.