The Circle of Kindness Foundation discussed the possibility of reducing the delivery period for Zolgensma from 50 to 25 days from the date of the blood test, according to a message on the Foundation’s VKontakte page.
“The time from a blood test to the administration of Zolgensma to the patient may take about 25 days rather than 50. The meeting participants outlined concrete steps aimed at accelerating delivery,” the message reads.
At the moment, it takes about 50 days to deliver the drug to an infant after a blood test taken as part of neonatal screening or in connection with suspected spinal muscular atrophy. The organization noted that the filing of application can be arranged in such a way that the Foundation will receive the application within two weeks after the blood test, and the expert council will hold emergency proceedings to consider the provision of Zolgensma.
Moreover, representatives of the Foundation discussed the issue of accelerating the supply of the drug more than twice – now, according to the contract, the manufacturer and supplier have to deliver within 25 days from the date when the expert council approves the application.
“Representatives of Skopinpharm, a company that supplies the drug to Russia, called such deadlines realistic, provided that it is possible to avoid flight cancellations when transporting the drug through third countries, since there are no direct flights between the US and Russia. As we found out, the production, delivery, and customs clearance themselves take less than 10 days,” the report says.
Zolgensma(onasemnogen abeparvovec), a therapy for SMA produced by Novartis, was registered in Russia in 2021.
Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus (AAV) vector containing a modified nucleic acid with a sequence of the SMN protein gene, which, when entering the nucleus of human motor neurons, restores the gene function lost due to mutation without embedding in human DNA, and restores the production of the SMN protein, which is responsible for motor neurons preservation and development. The drug penetrates the blood-brain barrier and reaches the motor neurons. Administered in the form of a single intravenous infusion, onasemnogene abeparvovec delivers a new functional copy of the SMN1 gene (transgene) into the patient’s cells, which stops the progression of the disease.
In accordance with the instructions for human use, the drug is intended for patients with SMA with a biallelic mutation in the SMN1 gene and a clinical diagnosis of type I SMA or patients with SMA with a biallelic mutation of the SMN1 gene and no more than three copies of the SMN21 gene.
The efficacy and safety of the drug was confirmed based on the results of four clinical trials.