- Biogen surprised the market Tuesday morning with much higher-than-expected earnings for its spinal muscular atrophy drug Spinraza (nusinersen).
- The drug brought in $203 million in sales — $30 million of which was due to inventory and $8 million of which was from sales outside the U.S.
- Spinraza, which was developed under an agreement with Ionis Pharmaceuticals, was approved by the Food and Drug Administration in late December.
The launch of Spinraza seems to be off to a strong start, but the drug has a loading dose that could mean sales growth could slow as fewer patients start therapy.
Spinraza requires four loading doses within the first two months — the first three doses spaced two weeks apart, with the fourth dose 30 days after — followed by one dose every four months thereafter. The drug costs about $125,000 per dose and therefore sales favor the beginning of the regimen.
Jefferies analyst estimate there are approximately 550 to 600 patients on Spinraza so far.
"We saw significant uptake in the U.S., and we are working with patients, physicians and payers to expand access to as many SMA patients as possible," said Biogen CEO Michel Vounatsos on a July 25 second quarter call. "And we are now building on this momentum outside the U.S. with recent approvals in Europe, Japan and Canada. We are preparing our launch efforts in these markets, and over time, we aim to make this therapy widely available across the globe."
There are now 145 sites that are administering Spinraza to patients, up from 88 in the first quarter.
"From a coverage and reimbursement perspective, we have seen strong progress towards expanding access," Vounatsos said.
The exec noted about 80% of commercially-insured lives in the U.S. now have a coverage policy in place for the drug. Meanwhile, about two-thirds of Medicaid patients have a formal policy.
There has been mounting pushback in the U.S. to pricey rare disease drugs. Some insurers have opted not to cover overly expensive drug for all patients while some pharmacy benefit managers have not been including certain drugs on their formulary lists.
Biogen noted there has been good uptake from both Type 2 and Type 3 SMA patients. "However, we believe that up to 60% of the Type 2 SMA population has already undergone some type of spinal surgery, creating challenges in intrathecal administration. As leading physician are transitioned to some of these more challenging cases, we expect slower uptake in Type 2 patients through the balance of the year," the CEO added.
The company noted it is working on new means of delivery for the drug and explained that as standards of care are updated, surgeons will have to perform surgeries that could allow for intrathecally administered therapy.
Biogen is also trying to promote ways to identify patients and has been pushing for more newborn screenings of the disease — at least one state has passed a bill regarding newborn screenings and four others are running pilot programs.
Outside of the U.S., where the drug has just gained approvals, Biogen estimates that there are about 12,000 patients.