Gene theraphy for Spinal Muscular Atrophy approved by FDA
- Dr. Kouzi
A new drug to treat spinal muscular atrophy (SMA) approved by the Food and Drug Administration (FDA) on Friday will come with a price tag of $2.1 million, making it the most expensive medicine in the world.
The drug, called Zolgensma, treats SMA, a genetic disease that causes debilitating muscle weakness and paralysis, and is a leading cause of infant mortality.
The high price tag is the latest illustration of how innovative drugs, while holding immense promise for improving the lives of the patients who need them, also pose a financial burden on the health care system through their cost.
The maker of the drug, Novartis, argued on Friday that the cost is still less than what it costs to treat people with SMA, which the company said was $4.1 million over 10 years.
"Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy,” said Vas Narasimhan, Novartis’s CEO. “Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system.”
A top FDA official said the drug provides the opportunity for improved care for people with SMA.
“Children with SMA experience difficulty performing essential functions of life. Most children with this disease do not survive past early childhood due to respiratory failure,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival.”
The drug will treat children under the age of two.
Treatment of Multiple Sclerosis with hematopoietic stem cells
- Dr. Kouzi
Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis A Randomized Clinical Trial
Richard K. Burt, MD1; Roumen Balabanov, MD2; Joachim Burman, MD3; et alBasil Sharrack, MD4; John A. Snowden, MD5; Maria Carolina Oliveira, MD6; Jan Fagius, MD3; John Rose, MD7; Flavia Nelson, MD8; Amilton Antunes Barreira, MD9; Kristina Carlson, MD10; Xiaoqiang Han, MD1; Daniela Moraes, MD6; Amy Morgan, APRN1; Kathleen Quigley, RN1; Kimberly Yaung, RN1; Regan Buckley, RN1; Carri Alldredge, RN1; Allison Clendenan, APRN1; Michelle A. Calvario, APRN1; Jacquelyn Henry, APRN1; Borko Jovanovic, PhD11; Irene B. Helenowski, PhD11
JAMA. 2019;321(2):165-174. doi:10.1001/jama.2018.18743
Question Is nonmyeloablative autologous hematopoietic stem cell transplantation (HSCT) more effective than disease-modifying therapy for patients with highly active relapsing-remitting multiple sclerosis (MS)?
Findings In this randomized clinical trial that included 110 patients with relapsing-remitting MS, treatment with nonmyeloablative HSCT compared with disease-modifying therapy resulted in a significantly prolonged time to disease progression (hazard ratio, 0.07).
Meaning In this preliminary study, nonmyeloablative HSCT was more effective than disease-modifying therapy for patients with relapsing-remitting MS.
Importance Hematopoietic stem cell transplantation (HSCT) represents a potentially useful approach to slow or prevent progressive disability in relapsing-remitting multiple sclerosis (MS).
Objective To compare the effect of nonmyeloablative HSCT vs disease-modifying therapy (DMT) on disease progression.
Design, Setting, and Participants Between September 20, 2005, and July 7, 2016, a total of 110 patients with relapsing-remitting MS, at least 2 relapses while receiving DMT in the prior year, and an Expanded Disability Status Scale (EDSS; score range, 0-10 [10 = worst neurologic disability]) score of 2.0 to 6.0 were randomized at 4 US, European, and South American centers. Final follow-up occurred in January 2018 and database lock in February 2018.
Interventions Patients were randomized to receive HSCT along with cyclophosphamide (200 mg/kg) and antithymocyte globulin (6 mg/kg) (n = 55) or DMT of higher efficacy or a different class than DMT taken during the previous year (n = 55).
Main Outcomes and Measures The primary end point was disease progression, defined as an EDSS score increase after at least 1 year of 1.0 point or more (minimal clinically important difference, 0.5) on 2 evaluations 6 months apart, with differences in time to progression estimated as hazard ratios.
Results Among 110 randomized patients (73 [66%] women; mean age, 36 [SD, 8.6] years), 103 remained in the trial, with 98 evaluated at 1 year and 23 evaluated yearly for 5 years (median follow-up, 2 years; mean, 2.8 years). Disease progression occurred in 3 patients in the HSCT group and 34 patients in the DMT group. Median time to progression could not be calculated in the HSCT group because of too few events; it was 24 months (interquartile range, 18-48 months) in the DMT group (hazard ratio, 0.07; 95% CI, 0.02-0.24; P < .001). During the first year, mean EDSS scores decreased (improved) from 3.38 to 2.36 in the HSCT group and increased (worsened) from 3.31 to 3.98 in the DMT group (between-group mean difference, −1.7; 95% CI, −2.03 to −1.29; P < .001). There were no deaths and no patients who received HSCT developed nonhematopoietic grade 4 toxicities (such as myocardial infarction, sepsis, or other disabling or potential life-threatening events).
Conclusions and Relevance In this preliminary study of patients with relapsing-remitting MS, nonmyeloablative HSCT, compared with DMT, resulted in prolonged time to disease progression. Further research is needed to replicate these findings and to assess long-term outcomes and safety.
Trial Registration ClinicalTrials.gov Identifier: NCT00273364
Stem Cell Transplantation to Treat Multiple Sclerosis