Spinal muscle atrophy is a hereditary disease in which all muscles, including those responsible for breathing, gradually atrophy. The most dangerous is the first type of SMA, which manifests itself even in infancy: about half of these patients do not live up to two years. This is the most common genetic cause of infant mortality.

743 minors with such a diagnosis living in Russia are registered in the register of the SMA Families Fund. Until recently, they had no chance to survive. But in 2019, the first drug was registered in the Russian Federation that helps stop the degeneration of alpha-motor neurons in the spinal cord. Spinrazoy therapy (nusinersen, manufactured by Biogen, distributors in the Russian Federation - Johnson & Johnson corporation Janssen group) should be life-long, and only the first course of injections costs about 40 million rubles.

Then it became known about the registration of the second drug in the United States - in the case of Zolgensma (AveXis production, Novartis group) one injection is enough, but its cost starts from 137 million rubles. In Russia, it has not yet been registered. The third medicine, Eurisdi (Risdiplam, manufactured by Roche), is also taken for life, its cost has not yet been determined, and it only goes through the registration process in the world.

RT spoke with representatives of drug manufacturing companies about the characteristics of these drugs and the prospects for their import into Russia.

- How many children have already received drug treatment in the world, including as part of pre-registration, that is, accelerated access for health reasons ? Is there any data on how many children from Russia are among them ?

Manufacturers of C Pinrase (C): As of December 31, 2019, more than 10 thousand patients were treated with Spinrase (nusinersen), including commercial patients, participants in the early access program, and participants in clinical trials.

Producers of “ Z olgenzma” (Z): We have covered nearly 400 families with gene therapy for spinal muscular atrophy (CMA), including as part of clinical trials and our managed access program in the USA, as well as on a commercial basis.

We also implement a program of free access on the basis of the lottery, which will allow the most fair distribution of doses. In 2020, a hundred people will receive treatment according to this scheme. Random selection of patients is carried out once every two weeks. We expect that the number of doses allocated will increase every six months.

Manufacturers of Eurisdi ( E ): Currently, in all clinical trials of the drug, more than 400 patients with SMA have received treatment or continue to receive it.

The global program for pre-registration access to the drug was launched on January 14, 2020. It is aimed at helping patients with the most severe manifestations of the disease, for whom registered medicines for SMA are contraindicated. The program will bring together 38 children with type 1 SMA from Russia. It is also now available for patients with type 2 SMA.

- Do you need special conditions in the clinics to conduct therapy with your drug?

C: In accordance with the instructions for use, the drug should be administered by doctors experienced in lumbar puncture.

Z: In order to ensure the safety of therapy for patients, it should be carried out in centers where the staff has undergone appropriate training. If there is no such center in the patient’s country of residence, he can undergo treatment in the country where he has it, either in the largest international medical center, or AveXis will evaluate the possibility of prompt training of the personnel of the medical center located in the patient’s country of residence.

E: The drug is available in powder form for the preparation of a solution for oral administration. After registration of the drug in the Russian Federation, relevant information on the conditions of preparation and storage of the solution will be available in the instructions for medical use.

- What is the cost of the drug made up of? Why is the treatment so expensive?

C: The pricing process aims to achieve an acceptable balance between the five key pricing factors. It should take into account the value of the drug for patients, the current and future benefits for society, keep in mind our desire for innovation, financing of further research and development. In addition, it is necessary to develop in the direction of value-oriented healthcare (optimizing the ratio of “treatment costs” / “result”. - RT ).

Z: In the USA, where the drug is approved, we used a value-oriented pricing system, as a result of which Solgensma costs 50% less than many other generally accepted treatment standards, including a ten-year course of SMA-therapy. In addition, the price of the medicine is in the range accepted by the Institute for Clinical and Economic Analysis of acceptable levels of profitability for drugs against rare diseases.

E: The price of a drug in the Russian Federation will become known after receiving a registration certificate and upon the availability of the drug on the Russian market. However, in January 2020, director of the pharmaceutical division of Roche, Bill Anderson, spoke about a possible pricing strategy for the drug (he said that the price of the drug would be lower than that of competitors, the same scheme was used when starting the drug for hemophilia. - RT ).

Unlike drugs designed to treat common diseases, orphan drugs are distinguished by high complexity and the associated high development cost. The cost may also be affected by the characteristics of the production process and logistics.

In general, Roche uses a value-oriented approach to pricing its medicines, which takes into account many factors, including medical need, the availability of alternative treatments, direct and indirect costs of medical care for the corresponding disease, the competitive situation, the level of economic development of the country and others.

- Where is the drug made now ? Will production expand, and if so, how exactly?

C: “Spinraza” is produced at several enterprises (depending on the production cycle). In Russia, each batch of product is marketed in accordance with the requirements of local regulations.

Z: Today, one of our enterprises has a license for the production of Solgensma. Two other businesses in the US states of Colorado and North Carolina are awaiting commissioning and licensing in 2020 and 2021, respectively.

E: Risdiplam is produced in Switzerland at two Roche plants in a full cycle - from the production of pharmaceutical substances to secondary packaging. At the moment, we do not have information about a possible expansion of production.

- What are the prospects for the drug in Russia? Will it become more affordable both in terms of price and, for unregistered drugs, in terms of legalization in the Russian Federation?

S: We are in dialogue with various stakeholders to ensure the widest possible access to the medicine.

Janssen now plans to file a dossier to consider the possibility of including nusinersen in the list of vital and essential medicines (Vital and Essential Medicines) in the spring of 2020, which implies that the price offered for registration will be lower than in reference countries.

Z: We applied for registration of the drug in almost three dozen countries, including Russia. At present, we cannot provide more detailed data on possible dates for registration.

E: In November 2019, the U.S. Food and Drug Administration (FDA) decided to prioritize the application for registering a diploma diploma. It is expected that the decision to register the drug in the United States will be made until May 24, 2020.

On March 18, we submitted an application to the Ministry of Health of the Russian Federation for the registration of Risdiplam drug (at the same time it was reported that the company plans to receive a registration certificate in the first half of 2021. - RT ).