Risdiplam

Mumbai|New Delhi: The commerce ministry and the ministry of health are in extensive deliberations over the issue of inclusion of data exclusivity or protection provisions in the proposed drug regulatory and legal framework, senior industry officials told ET. The matter has gained prominence in the last few weeks amid extensive lobbying by domestic drug industry groups, civil society, patient advocacy groups, and pharma multinationals. All the concerned parties made opposing claims to senior officials in the ministries on how data protection provisions could impact access to lifesaving drugs for Indian patients. "An inter-departmental secretary level meeting was held recently but there was no consensus," one of the officials said. "There is a positive indication on the acceptance of data protection in the regulatory provisions," a second official added. A third official termed it as an area of ongoing discussions, hinting a final decision has yet to be taken, and that the Centre may eventually explore a middle path, ensuring data protection for a specific term with certain caveats. The issue of data protection hit the spotlight recently after commerce minister Piyush Goyal, highlighted that the India-EFTA Trade and Economic Partnership Agreement (TEPA) entails an investment commitment of $100 billion over 15 years and the creation of one million jobs. The four members of the European Free Trade Association (EFTA) are Switzerland, Iceland, Norway, and Liechtenstein. The investment commitment from the four-nation bloc, officials said, is partly predicated on India's acceptance of data exclusivity or protection norms. Data exclusivity ensures protection of drug tests and other data originating from the research of an invention of drug or therapy by an innovator or originator company. While the matter pertains to the health ministry and the department of pharmaceuticals, the commerce ministry's Department for Promotion of Industry and Internal Trade (DPIIT) is also looking at it as the request came from EFTA. "The EFTA bloc has been requesting for a provision for regulatory data protection or the data which is generated as a result of clinical trials so as to encourage innovator companies and promote an innovative environment in India," a commerce ministry official said. "Preliminary discussions are going on," the official said, adding no proposals have been made by the Centre so far on the issue. To secure marketing approval for a new drug, pharma firms must submit the data or clinical trial results to the local regulator for its review process. However, global drug makers contend that the same data developed over years of rigorous trials and forming the basis of approvals is used by subsequent filers or generic drug makers who skip such trials or bioequivalence studies, securing an easy path to gaining regulatory approvals. This October, the Drug Controller General of India (DCGI) invited views on the "lack of level playing field", as the government intended to review data protection laws. According to a submission accessed by ET, the Organisation of Pharmaceutical Producers of India (OPPI), a lobby group comprising multinational pharma companies, has sought a provision of ten- year data protection for small molecules and 12 years for biological products. It stressed that this was in line with its European partners that grants ten-year data protection. "The government is considering a five-year data protection," said a senior industry official. However, Indian drug makers contend that data exclusivity is a layer of protection separate from patent rights. "Data exclusivity can block generic competition, delay competition, and price reduction, which would deprive patients of medicines at affordable prices," an official said. "A 1-3 year or 3-5 year data exclusivity period after an originator medicine is licensed is being considered," the person said. "Certain American companies have proposed a 10-12 year data protection period and the protection to start from the date when they apply for in India, not globally. European majors have sought around a 6-8 year exclusivity period". The Working Group on Access to Medicines and Treatments, a patient advocacy group, underlined that having data exclusivity in India would have delayed by several years the launch of affordable versions of Swiss drugmaker Roche's Risdiplam, used for treating spinal muscular atrophy (SMA), denying countless SMA patients access to a lifesaving treatment. "Data exclusivity will not be just a technical tweak; it will be a direct threat to the survival of many people," the group cited a patient as saying. India's Natco Pharma launched generic versions of Risdiplam at ₹15,900 per bottle, fraction of Roche's price of ₹6 lakh, following a favourable court verdict recently. By Vikas Dandekar , Kirtika Suneja & Teena Thacker ,

Like Zolgensma, Novartis' Itvisma is a one-time treatment that delivers a functional copy of the human SMN gene to treat SMA. Novartis has received an FDA approval for Itvisma, a new version of the company’s gene therapy Zolgensma, to treat older patients with spinal muscular atrophy (SMA).The approval comes more than six years after the FDA first cleared Zolgensma for SMA patients under 2 years old. Itvisma now covers all older patients with a confirmed mutation in the SMN1 gene.Both therapies work by delivering a functional copy of the human SMN gene via adeno-associated virus (AAV) vectors. While their active drug substances are identical, Itvisma is given directly to the central nervous system via an intrathecal injection in the spinal cord area, whereas Zolgensma is administered intravenously. Thanks to that formulation, Itvisma can be given to patients with larger weight in a more concentrated dose.Novartis is pricing Itvisma at a wholesale acquisition cost of $2.59 million, a company spokesperson told Fierce Pharma. At that price, the one-time therapy costs 35% to 46% less than existing chronically dosed treatments over the span of 10 years, according to the spokesperson.Patients’ out-of-pocket cost may vary, although Novartis expects broad coverage, the spokesperson added. The company is offering a patient support program in which eligible individuals may pay as little as $0.About 9,000 people in the U.S. live with SMA, and the majority of them are older children, teens and adults, Tracey Dawson, Ph.D., Novartis’ head of U.S neuroscience business, said in an interview with Fierce Pharma ahead of the approval.Given the broader patient population, Novartis has projected multibillion-dollar peak sales for Itvisma, also known as OAV101 IT. Since its initial approval in 2019, Zolgensma has treated more than 5,000 patients globally. Last year, the intravenous therapy brought in sales of $1.2 billion, making it the world’s best-selling gene therapy. Itvisma is geared toward patients who’re considering an option that may free them from the burden of chronic treatment, Dawson said. Novartis expects many of Itvisma’s recipients to be switching from existing therapies such as Biogen’s Spinraza and Roche’s Evrysdi, she said.Itvisma proved its worth in that population in the phase 3b Strength trial. Among 27 patients who discontinued treatment with either Spinraza or Evrysdi, their motor function, as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) score, showed stabilization over one year after receiving Itvisma. For the 66-point assessment, patients on Itvisma saw an increase in HFMSE least squares score of 1.05 by one year.The intrathecal gene therapy also works in treatment-naïve patients. In the Steer study, Itvisma showed a statistically significant 2.39-point improvement on the HFMSE, versus 0.51 points in patients who got a sham treatment.“While the numbers might not look huge, what it means from a clinical impact to a patient is that they’re able to continue to use their wheelchair controls, or they’re able to continue to walk,” Dawson said.Some of the seemingly tiny improvements, such as being able to keep grip strength, are meaningful to SMA patients, she said.To counter newer SMA options, Biogen has developed a higher-dose version of Spinraza. The new formulation just scored backing from the Committee for Medicinal Products for Human Use of the European Medicines Agency, setting up for a marketing authorization from the European Commission. Following a complete response letter in September related to certain technical information request, the FDA has accepted a resubmission from Biogen and has set a target decision date for high-dose Spinraza on April 3, 2026. Novartis is investing heavily in neuromuscular diseases lately. The Swiss pharma is shelling out about $12 billion to acquire Avidity Biosciences. The deal would give Novartis an RNA-based platform that produces antibody oligonucleotide conjugates, plus three late-stage muscular dystrophy programs.The deal follows another smaller acquisition a year ago of AAV gene therapy specialist Kate Therapeutics, which leverages in vivo expression of transgene RNA.