The approval of the drug will bring a new window of opportunity for the companies, both geographically and therapeutically
Published By: Eunice Gettys on September 28, 2016 07:31 am EST
Biogen Inc (NASDAQ:BIIB) and Ionis have been filling their mutual investigational molecule nusinersen for the New Drug Application (NDA) to the Food and Drug Administration (FDA). The molecule is indicated for the management and treatment of type-1 spinal muscular atrophy (SMA).
In August 2016, both the drugmakers have published their ENDEAR clinical study results in relation to the SMA type 1 disease with achievement of the primary endpoints. With reference to the results, the companies have shifted patients in an Open Label Extension Study from clinical trial. The investigational molecule has shown drastic improvement in the motor symptoms of patients receiving the drug.
Biogen has worldwide rights of the drug, for which it has paid $75 million to Ionis. The company has to pay other royalties for the clinical trials, regulatory approvals and marketing of the drug. In addition, Ionis is eligible to get $150 million in downstream payments in relation to the regulatory milestones.
MDA has provided $750,000 to Adrian Krainer at Cold Spring Harbor Laboratory in New York for the clinical research on the early stage patients of SMS to vitalize the role of nusinersen. Otherwise, MDA has invested approximately $45 million to ascertain and develop the role of nusinersen to look for other pathological aliments.
Moreover, the companies are planning for the submission of the drug to European Medicines Agency (EMA) in October 2016 and other geographical areas by the end of 2016. The EMA committee for Medicinal Products for Human Use (CHMP) has given approval for Accelerated Assessment reducing the standard protocol time.
Spinal muscular atrophy (SMA) is a debilitating disease affecting the patients via muscle wasting due to nerve cell degeneration. Approximately one in 10,000 live births are affected by this disease. According to Biogen’s statistics, there are 35,000 infant patients suffering from the debilitating disease across US, Europe, and Japan.
There is no treatment available till date for the disease and if approved, the drug can generate $1 billion on yearly basis. The molecule has already gained secured status due to its vitality in the US and Europe and is expected to gain approval from the regulatory agencies keeping in view of the important role it has to play.