EU Flags at the European Commission Building
A genetic disorder that slows bone growth and leads to dwarfism now has an approved drug, a biologic therapy developed by BioMarin Pharmaceutical. The European Commission decision announced Friday makes the drug, vosoritide, the first drug therapy for the rare disease, whose complications are typically addressed by surgery.
The disease, achondroplasia, is caused by a mutation to the gene that produces fibroblast growth factor receptor 3 (FGFR3), a protein that down-regulates bone growth. Activity of FGFR3 is typically kept in check by a peptide called C-type natriuretic peptide (CNP). BioMarins drug, given as a once-daily injection, is an analog of that peptide. The San Rafael, California-based rare diseases drug developer will market the drug under the name Voxzogo.
Achondroplasia is usually caused by a spontaneous mutation, according to the National Organization of Rare Disorders. In about 20% of patients, the condition is inherited. The condition is the most common cause of dwarfism. People who have achondroplasia have short limbs, leading to an average height of 4 feet. Potential complications include compression of the spinal cord, sleep apnea, bowed legs, and recurrent ear infections. The main treatments are surgeries to address spinal and limb problems.
BioMarin has only tested its drug in children spanning the age range when growth plates are still open. In an open-label dose-finding Phase 2 study enrolling 35 children 5 to 14, the annual growth velocity increased from baseline for all groups measured through 42 months. Results were published in 2019 in the New England Journal of Medicine.
The company proceeded to test the drug in a placebo-controlled Phase 3 study enrolling more than 120 children ages 5 to 14. Late last year, BioMarin reported that those in the open-label long-term extension of the Phase 3 study maintained an increase in annual growth velocity through the second year of continuous treatment. In children treated with the BioMarin drug, the cumulative height gain over the studys two years was 3.52 cm more than that of untreated children.
The extension study that is continuing to follow children who participated in the clinical trials has yet to reach the five-year mark. But BioMarin said that so far, the growth rates have been sustained and adverse events have been mild. The most common problems were injection site reactions and low blood pressure. BioMarin added that the adverse events reported did not include disproportionate bone growth or other bone problems, and there is no evidence so far of accelerated bone age or negative changes in bone mineral density.
According to BioMarin, an estimated 11,000 children in Europe, the Middle East, and Africa have achondroplasia and could be eligible for treatment with Voxzogo. The drug is still under FDA review. The U.S. agency has set Nov. 20 as the target date for a regulatory decision.
Incyte, Morphosys cancer drug gets European approval in lymphoma
In other European Commission drug approval news, Incyte and Morphosys were granted conditional marketing authorization for tafasitamab as a treatment diffuse large B-cell lymphoma (DLBCL). The decision for the antibody drug covers adults whose disease has relapsed or has not responded to earlier treatment, and who are not eligible for a stem cell transplant. Approval of the drug, which will be marketed in Europe under the name Minjuvi, covers its use in combination with Bristol Myers Squibb cancer drug lenalidomide, followed by Minjuvi as a monotherapy.
European approval of Minjuvi comes about one year after the FDA awarded accelerated approval to the drug. Incyte and MorphoSys share rights to the drug; the partners co-market the therapy in the U.S. under the name Monjuvi. Wilmington, Delaware-based Incyte holds exclusive commercialization rights to the drug outside of the U.S.
Photo: sinonimas, Getty Images
The rest is here:
BioMarin stands tall with first approved drug for rare disease that causes dwarfism - MedCity News
- SUM Hospital conducts Odishas first stem cell transplantation - The Times of India - April 24th, 2024
- Cancer treatment: What is stem cell therapy, and how does it work? - Moneycontrol - April 24th, 2024
- The murky, unregulated world of anti-ageing stem cell therapy - The Telegraph - April 24th, 2024
- Drug Prototype Promising in Treatment of Pulmonary Disease - Medscape - April 20th, 2024
- Man Paralyzed In Surfing Accident Regains Ability to Walk After Stem Cell Treatment - The Inertia - April 20th, 2024
- Medical breakthrough: 8-year-old boy first in New York to receive FDA-approved gene therapy for rare inherited blood disorder - WABC-TV - April 20th, 2024
- Stem Cell Treatment Helped A Man Who Was Paralyzed From The Neck Down To Stand On His Own - Bored Panda - April 20th, 2024
- UM, CUHK jointly hold symposium on stem cells and regenerative medicine - gcs.gov.mo - April 20th, 2024
- Paralysed patient now able to walk as stem cell therapy shows promising results - WION - April 15th, 2024
- Andrew Steele Sees Aging as the Root of Disease - Worth - Worth Magazine - April 15th, 2024
- The astonishing effect of stem cell implants - as groundbreaking treatment 'transforms' life for those with brain damage ... - Daily Mail - April 15th, 2024
- Consumer Alert on Regenerative Medicine Products Including Stem Cells - FDA.gov - April 13th, 2024
- This Startup Wants To Be OpenAI Of Stem Cell Therapy, Targets $250B Market - Forbes - April 13th, 2024
- Brain-cell transplants are the newest experimental epilepsy treatment - MIT Technology Review - April 3rd, 2024
- Global Regenerative Medicine Market to Touch Valuation of USD 472.95 Billion by 2032, at 25.86% CAGR: Astute ... - Yahoo Finance - April 1st, 2024
- Explosive Growth Projected for Stem Cell Market, Expected to Surpass $37 Billion by 2030 As Revealed In New... - WhaTech - April 1st, 2024
- Integrating New Therapies With Autologous Stem Cell Transplant in Myeloma - Targeted Oncology - April 1st, 2024
- New tool provides researchers with improved understanding of stem cell aging in the brain - Medical Xpress - March 30th, 2024
- The ISSCR announces 2024 election results - EurekAlert - March 28th, 2024
- Exosomes and Stem Cells Are the Future of Anti-Aging - NewBeauty Magazine - March 28th, 2024
- Charles River Extends Gene Therapy Alliance with NUS Medicine - Contract Pharma - March 22nd, 2024
- Exciting opportunities in stem cell technology and regenerative biology in Indian healthcare industry - Express Healthcare - March 22nd, 2024
- Charles River Announces Extension of Gene Therapy Manufacturing Alliance with NUS Yong Loo Lin School of Medicine - Yahoo Finance - March 22nd, 2024
- Opinion: The revolutionary sickle cell therapies - MSN - March 22nd, 2024
- 10 Years of Kansas Regenerative Medicine Center - KSAL - March 22nd, 2024
- BioCardia and StemCardia Announce Biotherapeutic Delivery Partnership - Diagnostic and Interventional Cardiology - March 17th, 2024
- Cynata Therapeutics' breakthrough in regenerative medicine - Finance News Network - March 17th, 2024
- New Positive Data Presented on Briquilimab Conditioning in Patients with Fanconi Anemia - GlobeNewswire - March 17th, 2024
- Novel 3D stem cell therapy to treat critical limb ischaemia - Drug Target Review - March 15th, 2024
- Breaking Boundaries: Pharmicell's Bold Vision for Healthcare Transformation and Monumental Growth - The Worldfolio - March 15th, 2024
- CuSTOM Organoid Research Evolving From Tool to Treatment - Research Horizons - Research Horizons - March 15th, 2024
- BioCardia and StemCardia Announce Partnership to Deliver Cell-Gene Therapy for Remuscularization of Heart - Cath Lab Digest - March 15th, 2024
- Calidi Biotherapeutics and City of Hope Announce Funding from the California Institute for Regenerative Medicine ... - Business Wire - March 13th, 2024
- Iron restriction keeps blood stem cells young, researchers find - Phys.org - March 13th, 2024
- Vitamin A could have a key role in both stem cell biology and wound healing: Study - Medical Dialogues - March 9th, 2024
- Trends in Stem Cell Transplantation Refusal for Myeloma Treatment - Targeted Oncology - March 7th, 2024
- Stem Cell Therapy Market (CAGR) of 31.1%, Future of Market Size Emerging Technologies and their Impact on ... - Taiwan News - March 7th, 2024
- This Swedish startup wants to reduce the cost, and controversy, around stem cell production - TechCrunch - March 6th, 2024
- Advancing stem cell therapy: The regulatory process to get to clinical trials - The Economic Times - March 6th, 2024
- Transitioning from traditional surgical methods to the innovative use of stem cells - pharmaphorum - March 6th, 2024
- 'Mini organs' are grown from human stem cells taken during the late stages of pregnancy for the first time - m - Daily Mail - March 6th, 2024
- Stem cell transplant cures man of HIV and leukemia, one of five to achieve full remission - Fox 28 - March 3rd, 2024
- Stem Cell Therapy Market to Boost USD 137.5 Billion by 2034 and Projected to grow at 32.10% CAGR with increasing ... - Yahoo Finance - March 1st, 2024
- Studies expand use of stem cell therapies - WPLG Local 10 - March 1st, 2024
- Innovative therapy targets and destroys leukemia stem cells - News-Medical.Net - February 27th, 2024
- Unlocking the Potential of Stem Cells: The Medical Revolution of the Century - EIN News - February 27th, 2024
- Pelage Pharmaceuticals Announces $16.75M Series A Financing led by GV to Revolutionize Regenerative Medicine ... - Yahoo Finance - February 27th, 2024
- Say Her Name - Henrietta Lacks, The Real Mother Of Stem - Her Campus - February 27th, 2024
- California Man Free of HIV And Cancer in Astonishing Medical Recovery - ScienceAlert - February 25th, 2024
- $93 million for research into improved health for children, young people and the aged, medical devices, mental health ... - Department of Health - February 25th, 2024
- Stem Cells in Regenerative Medicine - PMC - National Center for ... - February 24th, 2024
- The Promise and Reality of Stem Cell Therapy: What You Need to Know - Corporate Wellness Magazine - February 22nd, 2024
- Stem Cell Therapy: A Beacon of Hope for Patients with Untreatable Conditions - Corporate Wellness Magazine - February 22nd, 2024
- Leading Experts in Stem Cell Therapy: A Global Overview - Corporate Wellness Magazine - February 22nd, 2024
- Can Stem Cells Help Reverse the Effects of Aging? - Corporate Wellness Magazine - February 22nd, 2024
- Stem Cell Therapy for Cancer: Hope on the Horizon? - Corporate Wellness Magazine - February 22nd, 2024
- Stem Cells and Autism: A Closer Look at Innovative Treatments - Corporate Wellness Magazine - February 22nd, 2024
- Regrowth and Restoration: The Science Behind Stem Cell Therapy for Hair - Corporate Wellness Magazine - February 22nd, 2024
- Brain Recovery: The Role of Stem Cells in Stroke and Cerebral Palsy Treatments - Corporate Wellness Magazine - February 22nd, 2024
- Turning Back the Clock: The Science of Stem Cells in Anti-Aging - Corporate Wellness Magazine - February 22nd, 2024
- Insights into cellular therapies for cancer treatment - Drug Target Review - February 22nd, 2024
- The Role of Stem Cells in Rheumatoid Arthritis Recovery - Corporate Wellness Magazine - February 22nd, 2024
- Dr Ernesto Romeros vision transforms immunotherapy regenerative medicine into a global leader in stem cell treatment - Khaleej Times - February 22nd, 2024
- World-Renowned Doctors Leading the Way in Stem Cell Research and Treatment - Medical Tourism Magazine - February 20th, 2024
- Choosing the Best: A Guide to America's Premier Stem Cell Therapy Providers - Medical Tourism Magazine - February 20th, 2024
- Navigating Through the Possibilities of Stem Cell Treatment for Eye Disorders - Medical Tourism Magazine - February 20th, 2024
- Global Leaders in Stem Cell Therapy: Where Excellence Meets Innovation - Medical Tourism Magazine - February 20th, 2024
- Excellence in Stem Cell Treatment: How the US is Pioneering Global Healthcare Solutions - Medical Tourism Magazine - February 20th, 2024
- Clarifying Memory Loss Treatments: The Role of Stem Cells - Medical Tourism Magazine - February 20th, 2024
- Battling Inflammation: Stem Cell Therapy for Rheumatoid Arthritis - Medical Tourism Magazine - February 20th, 2024
- Digestive Health and Stem Cells: Treating Ulcerative Colitis and Crohn's Disease - Medical Tourism Magazine - February 20th, 2024
- Top Stem Cell Clinics in the USA: Pioneering Treatments for Chronic Conditions - Medical Tourism Magazine - February 20th, 2024
- Stem Cells and Autism: Understanding the Potential for Innovative Treatments - Medical Tourism Magazine - February 20th, 2024
- Stopping the awakening of sleeping Acute Myeloid Leukaemia stem cells to prevent disease relapse - University of Birmingham - February 20th, 2024
- City of Hope Achieves Ground Breaking Treatment for Blood Cancer and HIV - Medriva - February 18th, 2024
- Regenerative Medicine Market Size to Worth USD 95 Billion by 2032 - InvestorsObserver - February 18th, 2024
- North America Organoids Market Projected to Reach US$ 5.35 Billion by 2030, Riding on a CAGR of 21.5% - GlobeNewswire - February 18th, 2024
- Chinese team tests lung treatment that may be first to reverse COPD damage - South China Morning Post - February 15th, 2024
- Why leukemic stem cells not harmed by chemotherapy begin to grow and produce AML cells after treatment - Medical Xpress - February 15th, 2024
- Regenerative Medicine Market is Expected to Reach $49.0 Billion | MarketsandMarkets - Yahoo Finance - February 15th, 2024
Recent Comments