Spinraza (nusinersen) is a biologic drug indicated for the treatment of spinal muscular atrophy (or SMA). In 1Q17, Biogen’s (BIIB) Spinraza generated revenues of around $47 million. The US Food and Drug Administration (or FDA) approved Spinraza as the first therapy for SMA in December 2016. Spinraza has been given orphan drug designation in the European Union and the US. To know more about Biogen’s market access strategy for Spinraza, please read Biogen Has Developed a Robust Access Strategy for Spinraza.
The revenue growth of Spinraza may positively impact the share prices of the Vanguard Health Care ETF (VHT). Biogen holds ~1.7% of VHT’s total portfolio holdings. Peers in the neurology drug market include Pfizer (PFE), Merck (MRK), and Amgen (AMGN), which account for 5.6%, 5.0%, and 3.5% of VHT’s total portfolio holdings, respectively.
The above graph represents Spinraza’s revenue trajectory from its launch in 4Q16 to 1Q17. To know more about Biogen’s marketing strategy for Spinraza, please read Biogen’s Targeted Marketing Strategy for Spinraza in 1Q17.
About ENDEAR phase three trial
The ENDEAR phase three clinical trial was conducted in infantile-onset SMA patients. The infantile SMA patients were administered Spinraza, and the drug demonstrated significant improvement in motor function compared to untreated patients. The study was conducted in over 170 patients. The drug received FDA approval after the study concluded. Around 40% of patients who received the drug achieved the motor milestone response compared to 0% for untreated patients. Spinraza achieved its primary endpoint in the study by demonstrating a substantial 47% reduction in permanent ventilation/risk of death. The end of study (or EOS) analysis showed that 68% of untreated infants with SMA died or had to be put in permanent ventilation compared to 39% of infants with SMA treated with Spinraza. Since Spinraza is currently the first drug for infantile SMA in the case of reduced access constraints, the revenue growth can be rapid for this drug.