EMA Signs Off on Conditional Authorization for Novartis Gene Therapy

Regulatory NewsRegulatory News | 27 March 2020 |  By 

The European Medicines Agency’s Committee for Advanced Therapies on Friday announced that it has recommended a conditional authorization for Novartis’ gene therapy Zolgensma (onasemnogene abeparvovec) as a treatment for babies and young children with spinal muscular atrophy (SMA), a rare and often fatal genetic disease.

EMA’s recommendation is based on the preliminary results of one completed clinical trial and three supporting studies in patients with SMA with different stages of disease severity. The trial enrolled 22 patients who were less than six months of age at the time of the gene replacement therapy with Zolgensma. EMA said that out of the 22 patients, 20 (91%) were alive and did not need permanent ventilatory support at 14 months of age.

Additional efficacy and safety data are being collected through three ongoing studies, a long-term registry and further investigations. EMA says that all results must be included in post-marketing safety reports, which are continuously reviewed by the agency.

Zolgensma was developed by AveXis, which was acquired by Novartis in an $8.7 billion deal in April 2018, and was approved by the US Food and Drug Administration in May 2019.

Nitrosamine Risk Extension

Meanwhile, the EMA also agreed to extend the deadline for marketing authorization holders to complete their risk evaluations on nitrosamine impurities until 1 October 2020. If a risk of presence of nitrosamines is identified as a result of the risk evaluation, confirmatory testing should be carried out, EMA says.

The risk evaluations are being carried out because in June 2018, EU authorities became aware of the presence of a nitrosamine, N-nitrosodimethylamine (NDMA), in valsartan from one manufacturer of active pharmaceutical ingredients (APIs). Subsequently, another nitrosamine, N-nitrosodiethylamine (NDEA), was detected and other sartans from more API manufacturers were later implicated. NDMA and NDEA are classified as probable human carcinogens.

“MAHs should work with manufacturers of API and finished products in order to review the API and finished product manufacturing processes with respect to the arrangements for preventing nitrosamine formation as well as contamination or cross-contamination, taking into account their knowledge of the manufacturing processes as well as the potential sources of nitrosamine impurities,” the UK’s MHRA said.

Register COVID-19 Studies

The EMA and the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) encouraged all researchers to register their pharmacoepidemiological studies related to the COVID-19 pandemic in the EU PAS Register.

“They should also upload and make their study protocols public, with a description of the data collected or planned to be collected. This is to facilitate collaborations and speed up the design of observational studies by others. Researchers should include ‘COVID-19’ in the study title to allow easy retrieval of all COVID-19-related studies via the ‘Title of Study’ search filter,” EMA said.

Data Errors

In addition, the EMA said Friday that a reassessment of Novartis’ breast cancer treatment Tyverb (lapatinib) did not resolve previous concerns and the product information will continue to state that no data are available on the effectiveness of Tyverb used together with an aromatase inhibitor compared with trastuzumab used with an aromatase inhibitor in patients previously treated with trastuzumab.

Data submitted in July 2018 indicated a benefit of Tyverb over trastuzumab when each medicine was used with an aromatase inhibitor. But in April 2019, errors were detected in the data and they were removed from the product information.

“During a procedure to reassess the data, the contract research organisation where the data were analysed was inspected. The inspection found deficiencies in the systems and procedures for managing data and concluded that data handling did not comply fully with good clinical practice (GCP). In addition, the re-analysed data do not allow a conclusion on whether Tyverb is more effective than trastuzumab when either is combined with an aromatase inhibitor,” EMA said.

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 23-26 March 2020


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