Industry Insights April Edition
Virica Biotech
Achieve higher yields, improve quality & scalability with Viral Sensitizers (VSE?)
?
???????????????????????????????
Report Overview:
Virica's Insights
The cell and gene therapy (CGT) industry continues to redefine the treatment landscape, offering curative solutions to previously untreatable diseases. 2023 is considered a breakthrough year for approvals, with seven FDA approvals in the US and one in the EU. With Q1 2024 behind us, there is sustained momentum with 2 new FDA approvals and 1 EMA approval, totalling 36 CGTs for the US and 19 for the European markets. The wave of approvals reflects the growing confidence of regulatory authorities; for example, just recently FDA approved J&J and Legend’s Carvykti for second-line therapy in multiple myeloma, significantly improving patient eligibility and accessibility. This implies an increase in demand as manufacturing strains become more pronounced.
Another shortfall is the exorbitant manufacturing costs of CGTs that impede patient affordability. Orchard Therapeutics recently approved gene therapy Lenmeldy to treat MLD in children is?priced at a staggering $4.25 million per dose, making it the most expensive drug on the market and highlighting cost pressures. It joins the ranks of?several approved CGTs with exorbitant price tags.?Phacilitate’s recent Advanced Therapies Week emphasized a need to reduce manufacturing costs through?automation, digitization and transition to more allogenic and “off-the-shelf” solutions?for cell therapy. A good example is?Ori Biotech’s pioneering efforts through its Lightspeed Early Access Program?(LEAP), which demonstrates the integration of bioreactor systems, automation and real-time data analysis, enhancing scalability and standardization efforts. Parallelly, there is an acute need?to improve the manufacturing productivity of viral vectors such as rAAV, which plays a pivotal role in CGT but suffers manufacturing bottlenecks.
The manufacturing constraints signal a potential?shift in the 2024 industry focus to manufacturability and scalability?to ensure groundbreaking therapies reach a broader patient base. Ultimately, CGT manufacturing is complex and needs solutions to tackle the challenge from multiple angles using complementary and synergistic technologies.
Top Headlines:
World’s Most Expensive Drug: Orchard’s Lenmeldy Priced at $4.25M US.
Orchard's newly FDA-approved therapy, Lenmeldy, is priced at $4.25M US, making it the highest-priced gene therapy on the market. The pricing determined by the ICER set Lenmeldy’s health benefit price benchmark up to $3.94M US and was found to have the highest value-based price for any treatment to date. Lenmeldy targets metachromatic leukodystrophy (MLD), a rare genetic metabolic disorder caused by a mutation in the ARSA gene, leading to toxic build-up. The treatment is curative with a single treatment. Orchard’s CEO Bobby Gaspar stated Lenmeldy is a “paradigm-shifting medicine.”
领英推荐
FDA Approves First Cell Therapy to Treat Metastatic Melanoma.
The FDA approved Iovance Biotherapeutics, Inc.’s Amtagvi, one-time autologous cell therapy and the first tumour-infiltrating lymphocyte (TIL) therapy to treat aggressive melanoma. “The approval of Amtagvi represents the culmination of scientific and clinical research efforts leading to a novel T cell immunotherapy for patients with limited treatment options,” said Dr. Peter Marks, director of FDA’s CBER.
CAR-T Faces Infrastructure and Manufacturing Reality Check.
Despite impressive efficacy and a growing market, the CAR-T industry currently faces high prices, small patient pools, and limited manufacturing capacity, constraining widespread adoption. Solving the infrastructure bottleneck requires a long-term collaborative approach involving healthcare providers, drug manufacturers, and regulatory agencies.
News at a Glance
?
?
?
?
?