The Ministry of Health has included Zolgensma in the pharmaceutical benefit of the National Health System (SNS), a new gene therapy treatment for the treatment of certain serious types of the disease called Spinal Muscular Atrophy (SMA) for the treatment of patients with SMA type 1 and presymptomatic.
It is estimated that around 30 pediatric patients a year will benefit from this neurodegenerative disease that affects the motor neurons of the anterior horn of the spinal cord; an autosomal recessive genetic disorder that is considered a rare disease, and that represents the most frequent genetic cause of infant mortality in children under two years of age. A problem that weakens the muscles in such a way that the patient is affected from speech to their ability to speak or swallow. According to data from the FundAME foundation, it is estimated that there are between 800 and a thousand people in Spain who suffer spinal muscular atrophy (SMA).
The new drug Zolgensma, which It is considered “the most expensive medicine in the world”, with a cost of 1.95 million euros per injection, it is administered intravenously in a single dose, its active ingredient is Onasemnogén abeparvovec. It is a gene therapy drug that manages to increase the expression levels of the SMN protein in motoneurons with its administration, which represents a new approach to the cause of this monogenic disease. As Vicente Arocas, from the Spanish Society of Hospital Pharmacy (SEFH), explains in El País, “the drug is administered with a single intravenous injection that introduces the gene and makes it possible for the body to alleviate this deficiency.”
Onasemnogene abeparvovec is the first gene therapy drug included in the pharmaceutical benefit for the treatment of presymptomatic SMA type 1 patients. This is the second medicine for the treatment of SMA patients, after the inclusion of the medicine whose active ingredient is nusinersen in March 2018.
Treatment should be initiated and administered in a healthcare setting and supervised by a physician experienced in the management of SMA patients.
Financed by the SNS from December 1
With the financing of this new gene therapy drug, Spain is among the European Union countries with the largest number of advanced therapy drugs financed by public health systems.
The public funding will be effective from December 1 and its results will be measured through VALTERMED, the SNS’s corporate information system for measuring health outcomes in the use of medicines.
The pricing of this drug was agreed upon by the Interministerial Commission on Drug Prices (CIPM) at the meeting held on October 28, 2021 and is based on a mixed financing model made up of a payment-by-results agreement and a volume payment agreement.
Likewise, some eligibility criteria have been agreed upon that identify patients in whom clinical benefit is expected, based on the evidence from clinical trials, that is, both improvement in quality and life expectancy of patients. that they receive it.
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