PUTRAJAYA: The 393rd Drug Control Authority (PBKD) meeting yesterday approved the registration of a cell and gene therapy product (CGTP) called Zolgensma for the first time since the control of the product came into force on Jan 1, 2021.
Health director-general Datuk Dr Muhammad Radzi Abu Hassan (pix) said the approval was granted after spinal muscular atrophy (SMA) was classified as a rare disease in Malaysia and Zolgensma, which is used to treat the disease, was designated an orphan medicine.
“This qualifies Zolgensma’s products for priority evaluation by the ministry’s National Pharmaceutical Regulatory Agency (NPRA) to provide immediate access to patients in need,“ he said in a statement today.
Zolgensma is a gene therapy based on an adeno-associated viral vector for the treatment of children below two years old with SMA.
SMA is a rare neuromuscular disease that is inherited, resulting in physical weakness and can lead to paralysis.
Dr Muhammad Radzi said that Zolgensma is manufactured by Novartis Gene Therapies, Inc. in Durham, United States and Novartis Corporation (Malaysia) Sdn Bhd is the product registration holder (PRH).
He said the meeting also approved a conditional registration approval for the QDENGA® dengue vaccine for the prevention of dengue fever in persons aged four years and above.
He said the product was approved after the PBKD was satisfied with the evaluation results on the efficacy, safety and quality of the product as well as the post-registration surveillance activities conducted by PRH.
Under this conditional registration, the PRH is required to submit surveillance data on the efficacy and safety of the QDENGA® vaccine from time to time after it has been used in the Malaysian market, he said.
“This allows the NPRA to continuously monitor both aspects to ensure that the benefit-risk ratio of the QDENGA® vaccine remains positive,“ said Dr Muhammad Radzi.
The QDENGA® vaccine is manufactured by Takeda GmbH in Germany, and Takeda Malaysia Sdn Bhd is the PRH for the product.
According to him, the government is committed to various efforts to improve people’s access to quality, safe and effective medicines. –Bernama