Major Approvals in Cell and Gene Therapies in Early 2024

Cell and gene therapy (CGT) is an innovative treatment strategy that promises the potential for a "one-time treatment, permanent cure," offering many patients the possibility of long-term relief from their medical conditions. In the first half of 2024, eight new CGT therapies were approved globally, marking significant advancements in the field.

1. Sarepta Therapeutics' Elevidys: On June 20th, Sarepta Therapeutics announced that the FDA had expanded the label for its gene therapy Elevidys, which treats Duchenne muscular dystrophy (DMD). Initially approved for pediatric patients aged 4-5 years, Elevidys is now approved for all patients aged 4 and above, regardless of their ambulatory status. This milestone includes regular approval for ambulatory patients and accelerated approval for non-ambulatory patients. The ongoing ENVISION study will further verify Elevidys' clinical benefits for non-ambulatory patients.

2. Geron's Rytelo: Approved on June 6th, Geron's Rytelo became the first FDA-approved oligonucleotide telomerase inhibitor. It treats transfusion-dependent anemia in lower-risk myelodysplastic syndromes (LR-MDS) patients. Its approval was based on successful Phase 3 IMerge trial results, showing significant reductions in red blood cell transfusion needs. This approval ends a near 40-year pursuit of telomerase-targeted therapy, and Rytelo's projected sales could reach $737 million by 2028.

3. Moderna's mRESVIA Vaccine: Moderna's mRNA respiratory syncytial virus (RSV) vaccine, mRESVIA, was approved by the FDA on May 31st for adults aged 60 and above. This is the first mRNA vaccine approved for a non-COVID-19 disease. The approval was based on a Phase 3 trial with around 37,000 participants, showing the vaccine's 83.7% efficacy against RSV lower respiratory tract disease. The study found no serious safety concerns and the vaccine's protective effects persisted over an 8.6-month follow-up.

4. Pfizer's BEQVEZ Gene Therapy: On April 26, 2024, Pfizer's BEQVEZ, an AAV vector-based gene therapy, received FDA approval for treating adults with moderate to severe hemophilia B. BEQVEZ significantly reduced the annualized bleeding rate in patients, as shown in the Phase III BENEGENE-2 study. It is priced at $3.5 million and is under review by the European Medicines Agency. Pfizer is also investigating other gene therapy programs for hemophilia A and DMD.

5. Orchard Therapeutics' Lenmeldy: On March 18, the FDA approved Orchard Therapeutics' Lenmeldy for treating metachromatic leukodystrophy (MLD) in pediatric patients. Priced at $42.5 million, Lenmeldy is the most expensive drug ever approved and the first FDA-approved therapy for early-onset MLD. The approval was based on data from a study involving 37 children, showing improved survival and reduced motor dysfunction.

6. Bristol Myers Squibb's Breyanzi: On March 14, the FDA granted accelerated approval to BMS's Breyanzi for treating relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). This CAR-T cell therapy targets CD19 and has shown a complete remission rate of 20% in the TRANSCEND CLL 004 trial. Breyanzi's unique composition helps manage cell therapy toxicities effectively.

7. Carsgen Therapeutics' Selinexor Injection: Approved by China's NMPA on March 1st, Carsgen Therapeutics' Selinexor Injection treats adult patients with relapsed or refractory multiple myeloma. Priced at 1.15 million RMB, it is the fifth CAR-T therapy approved in China. The therapy uses a lentiviral vector for high binding affinity and stability and is undergoing further trials in North America.

8. Iovance Biotherapeutics' Amtagvi: On February 16th, Iovance Biotherapeutics' Amtagvi received FDA accelerated approval for treating advanced melanoma. Priced at $515,000, Amtagvi is the first approved tumor-infiltrating lymphocyte (TIL) therapy. Clinical data showed a 31.4% objective response rate, with 8 complete responses and 40 partial responses.

Looking ahead, up to five other CGTs might be approved in 2024, including therapies from Carsgen, Adaptimmune, BioCardia, Pfizer, and Autolus. However, recent rejections by the FDA for Rocket Pharmaceuticals' and Abeona Therapeutics' therapies highlight ongoing challenges in the field.

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