NEW YORK (Reuters) - Novartis is in discussion with patient groups over its lottery-style free drug program for its multi-million-dollar gene therapy for spinal muscular atrophy (SMA) after criticism that the process could be unfair to some babies with the deadly disease.
FILE PHOTO: The company's logo is seen at the new cell and gene therapy factory of Swiss drugmaker Novartis in Stein, Switzerland, November 28, 2019. REUTERS/Arnd Wiegmann
The company said on Friday that it will be open to refining the process in the future, but it is not making any changes at this time. The program is for patients in countries where the medicine, called Zolgensma, is not yet approved for the rare genetic disorder, which can lead to death and profound physical disabilities.
At $2.1 million per patient, Zolgensma is the worlds costliest single-dose treatment.
Novartis said the program will open for submission on Jan. 2 and the first allocation of drugs would begin in February. Novartiss AveXis unit, which developed the drug, will give out 50 doses of the treatment through June for babies under 2 years old, it said on Thursday, with up to 100 total doses to be distributed through 2020.
Patient advocacy group SMA Europe had a conference call with the company on Friday, according to Kacper Rucinski, a board member of the patient and research group who was on the call.
There are a lot of ethical questions, a lot of design questions that need to be addresses. We will be trying to address them in January, Rucinski said. He said the program has no method of prioritizing who needs the treatment most, calling it a Russian roulette.
The company said it developed the plan with the help of bioethicists with an eye toward fairness.
This may feel like youre blindly passing it out, but it may be the best we can do, said Alan Regenberg, who is on the faculty at Johns Hopkins Berman Institute of Bioethics and was not among the bioethicists Novartis consulted with on the decision. It may be impossible to separate people on the basis of prognosis out of the pool of kids under 2, he said.
According to Rucinski, the parties will continue their discussion in January to see what can be improved in the design of the program.
Novartis said on Thursday that because of manufacturing constraints it is focused on providing treatment to countries where the medicine is approved or pending approval. It has one licensed U.S. facility, with two plants due to come on line in 2020.
Zolgensma, hit by turmoil including data manipulation allegations and suspension of a trial over safety concerns, is the second SMA treatment, after Biogens Spinraza.
Not all of the SMA community are opposed to Novartis program.
Rajdeep Patgiri moved from the United Kingdom to the United States in April so his daughter could receive Zolgensma. She has responded well to the treatment, and Patgiri worries that negative attention to the program could keep patients from receiving the drug.
The best outcome for all patients would be if everybody could get the treatment. Given all the constraints, a lottery is probably the fairest way to determine who receives the treatment, he said.
Reporting by Michael Erman; Additional reporting by John Miller in Zurich; Editing by Leslie Adler
- gene therapy | Description, Uses, Examples, & Safety ... - February 24th, 2020
- Gene therapy - Mayo Clinic - February 24th, 2020
- How does gene therapy work? - Genetics Home Reference - NIH - February 24th, 2020
- Nationwide Childrens among hospitals leading the way in gene therapy - The Columbus Dispatch - February 24th, 2020
- BioMarin gene therapy won't need an AdComm as it nabs speedy FDA review - FierceBiotech - February 24th, 2020
- Gene therapy to halt rare form of sight loss - BBC News - February 24th, 2020
- With supply tight, Novartis readies gene therapy plant for production - BioPharma Dive - February 24th, 2020
- New Gene Therapies Institute Aims to Address Regulatory Barriers - HealthITAnalytics.com - February 24th, 2020
- Bluebird's gene therapy hits another delay, this time in the US - BioPharma Dive - February 24th, 2020
- First patient undergoes Luxturna gene therapy on NHS - PharmaTimes - February 24th, 2020
- First Patients Begin Gene Therapy Treatment for Blindness through NHS - Interesting Engineering - February 24th, 2020
- Spark R&D chief High exits in wake of Roche takeover - FierceBiotech - February 24th, 2020
- Why So Many Biotechs Are Scrambling to Develop a Drug for the Same Rare Disease - The Motley Fool - February 24th, 2020
- 3 Cancer Treatment Stocks to Buy Right Now - The Motley Fool - February 24th, 2020
- The Most Important Stock to Own Over the Next Decade - Nasdaq - February 24th, 2020
- FDA Continues Strong Support of Innovation in Development of Gene Therapy Products - FDA.gov - February 2nd, 2020
- Interpreting Sameness Gene Therapy Products Orphan Drug Regulations - FDA.gov - February 2nd, 2020
- Pfizer lays out gene therapy aspirations - BioPharma Dive - February 2nd, 2020
- Global Gene Therapy Industry Outlook 2020-2024 - Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities - P&T Community - February 2nd, 2020
- FDA guidance on gene therapies development and manufacturing - BioPharma-Reporter.com - February 2nd, 2020
- Devastation, then hope: Platteville baby first in state treated for rare condition after screening - Madison.com - February 2nd, 2020
- Studies target unilateral gene therapy injection - Ophthalmology Times - February 2nd, 2020
- Otonomy Presents Preclinical Results for GJB2 Gene Therapy Collaboration and Cisplatin Otoprotection Program - Yahoo Finance - February 2nd, 2020
- Eyeing a trio of trial initiations, Jim Wilson's gene therapy startup woos Bruce Goldsmith from Deerfield as CEO - Endpoints News - February 2nd, 2020
- The FDA unveils a new regulatory framework to speed along gene therapies, rewarding the leading players - Endpoints News - February 2nd, 2020
- Gene Therapies Make it to Clinical Trials - Discover Magazine - December 31st, 2019
- Experimental Gene Therapy Shows Promise for Preventing and Treating Lou Gehrig's Disease in Mice - BioSpace - December 31st, 2019
- 2019: The year gene therapy came of age - INQUIRER.net - December 31st, 2019
- BLA Submitted for Gene Therapy to Treat Hemophilia A - Monthly Prescribing Reference - December 31st, 2019
- First Alzheimer's Disease Gene Therapy Human Study Provided by Maximum Life Foundation, Offers 10 Free Therapies for Qualifying Patients - Benzinga - December 31st, 2019
- Year in Review: Gene Therapy Technology and a Milestone 2019 for Medical Research - News18 - December 31st, 2019
- Gene therapy to conquering hepatitis C: A decade of medical breakthroughs - Business Standard - December 31st, 2019
- What were the biggest biopharma stories of 2019? - MedCity News - December 31st, 2019
- Global Gene Therapy Market 2019 Revenue, Opportunity, Forecast and Value Chain 2025 - Market Research Sheets - December 31st, 2019
- Manufacturing: the next breakthrough in gene therapy - STAT - December 23rd, 2019
- Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options - Xconomy - December 23rd, 2019
- Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It's Riddled With Controversy - Singularity Hub - December 23rd, 2019
- Making advanced therapies takes industrializing personalization - STAT - December 23rd, 2019
- Dyno Therapeutics Launches to Improve Viral Vectors for Gene Therapy - BioSpace - December 23rd, 2019
- New Gene Therapy Method May Open BRAVE New World in Parkinson's - Parkinson's News Today - December 23rd, 2019
- Pharma's gene and cell therapy ambitions will kick into high gear in 2020despite some major hurdles - FiercePharma - December 23rd, 2019
- Viewpoint: EU should take a lead in enforcing the corporate social responsibility of gene therapy manufacturers - Science Business - December 23rd, 2019
- Takeda Presents Data for Hemophilia A and B Gene Therapy Optimization - Hemophilia News Today - December 23rd, 2019
- GenScript Biotech to Host Global Forum on Cell and Gene Therapy and the Booming China Market During JPM Week - PRNewswire - December 23rd, 2019
- The gene therapy research that could save a family of four - News - The University of Sydney - December 23rd, 2019
- Waning treatment is a warning for all 'one-and-done' therapies - STAT - December 23rd, 2019
- Would you pay $1M to partake in an anti-aging gene therapy trial? - Fox Business - December 13th, 2019
- Pfizer to bring gene therapy production in-house - BioPharma-Reporter.com - December 13th, 2019
- Buyer beware of this $1 million gene therapy for aging - MIT Technology Review - December 13th, 2019
- Dr. James Wilson, a scientific pioneer, on the future of gene therapy - STAT - December 13th, 2019
- The Rise Of Patent Wars In Europe's Gene Therapy Space - Law360 - December 13th, 2019
- Pfizer cites competitiveness of inhouse gene therapy - Bioprocess Insider - BioProcess Insider - December 13th, 2019
- Investment in UK cell and gene therapy manufacturing set to continue - European Pharmaceutical Review - December 13th, 2019
- Biomanufacturing and Supply Chain Standardization Key to Success in Cell and Gene Therapy Industry Boom - BioBuzz - December 13th, 2019
- Research targets gene therapy for exudative AMD patients - Modern Retina - December 13th, 2019
- Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug... - December 13th, 2019
- GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery - Business Wire - December 13th, 2019
- First 2 Adults with Severe Hemophilia A Respond Well to Gene Therapy BAY 2599023 in Clinical Trial - Hemophilia News Today - December 13th, 2019
- New Anti-Aging Clinical Trial Begins. For $1 Million, You Can Be a Participant. - Livescience.com - December 13th, 2019
- Watch out, Keytruda. Ferring's bladder cancer gene therapy rival has new dataand they look competitive - FiercePharma - December 13th, 2019
- Solid Biosciences Stock Crashes Over Safety Concerns About Its Gene Therapy - Barron's - November 13th, 2019
- Solid's Duchenne gene therapy trial halted after patient suffers toxicity - STAT - November 13th, 2019
- Gene Therapy in Neurology: 2019 Overview & Forecast Report - Yahoo Finance - November 13th, 2019
- How Gene Therapy Is Evolving to Tackle Complex... - Labiotech.eu - November 13th, 2019
- Lonza taps Cryoport to bolster cell and gene therapy delivery - BioPharma Dive - November 13th, 2019
- Pfizer's 'brainstorming' payment deals as gene therapies advance, exec tells Bloomberg - FiercePharma - November 13th, 2019
- Next generation cell and gene therapies: fine tuning the promise - Business Weekly - November 13th, 2019
- Vertex invests in gene therapy manufacturing - BioPharma-Reporter.com - November 13th, 2019
- Scientists are using gene therapy to treat a heart disease in dogs. Could humans be next? - 10News - November 13th, 2019
- Updated Alta Trial Results Support SB-525 Gene Therapy for Hemophilia A - Hemophilia News Today - November 13th, 2019
- Sangamo Announces Gene Therapy and Ex Vivo Gene-Edited Cell Therapy Data Presentations at the American Society of Hematology Annual Meeting - Business... - November 13th, 2019
- Modified Protein Enhances the Accuracy of CRISPR Gene Therapy - DocWire News - November 13th, 2019
- Triple-Gene Announces Completion of Enrollment and Dosing in Phase 1 Trial of INXN4001, First Multigenic Investigational Therapeutic Candidate for... - November 13th, 2019
- Five benefits of gene therapies - Echo Live - September 19th, 2019
- Reprogramming the Human Computer: Silicon Valley Meets Cell and Gene Therapy - BioBuzz - September 19th, 2019
- FDA approves 2nd gene therapy cancer drug from Durham's Precision Bio for clinical trial - WRAL Tech Wire - September 19th, 2019
- 7-Year-Old Receives New FDA-Approved Retina Gene Therapy - University of Michigan Health System News - September 19th, 2019
- First Patient Dosed in Trial of Fabry Gene Therapy Candidate FLT190 - Fabry Disease News - September 19th, 2019
- Bluebird bio reveals further encouraging data for CALD gene therapy - PMLiVE - September 19th, 2019
- Joseph M. Sanzari Childrens Hospital and John Theurer Cancer Center Launch Clinical Trial Evaluating Gene Therapy for Severe Sickle Cell Disease in... - September 19th, 2019