Biogen and Ionis Pharmaceuticals are moving quickly with their designer therapy for spinal muscular atrophy (SMA), a debilitating genetic disorder that affects approximately one in 10,000 newborns in the United States.
The companies announced Monday positive interim results from a Phase 3 trial of Spinraza (nusinersen) for Type 2 (later-onset) SMA. The trial will now be shelved so all patients can receive the active compound.
When Investment Rhymes with Canada
Canada has a proud history of achievement in the areas of science and technology, and the field of biomanufacturing and life sciences is no exception.
In a phone interview, Sarah Boyce, VP and chief business officer at Ionis (formerly Isis Pharmaceuticals), said the drug represented science at its best.
“When you understand a disease and exactly what’s driving it, you can create a targeted drug that is potentially transformational,” said Boyce. “This was very precisely designed.”
SMA is a degenerative motor neuron disease caused by a defect or deletion in the survival motor neuron 1 (SMN1) gene. Patients with the condition progressively struggle with voluntary movement and muscle control.
SMA is considered the leading cause of infant death in the United States, but no disease-modifying therapies are currently approved.
Yet many companies, including Progenity, GenPath, and Quest Diagnostics, all offer carrier screening for SMA. This involves testing one or both parents, ideally before conception, to understand if their future offspring are at risk of developing the disease.
If both parents are deemed carriers of the SMN1 mutation, different fertility options such as IVF, use of a surrogate, or adoption, may be considered. Routine carrier screening therefore, has the potential to decrease the number of children born with the disorder.
When asked about the future of the patient population, Boyce said market projections were now in the hands of Biogen, who did not respond to email or phone requests for an interview.
Boyce did point out that the therapy has the potential to increase lifespans, which would help sustain demand for the drug. Spinraza has orphan disease status in both the U.S. and EU.
For current patients and their families, effective treatments are front-of-mind. Boyce said the efforts of the SMA community were a vital part of the trial’s success.
According to the companies’ news release, the FDA has accepted their New Drug Approval (NDA) request with an aim to act quickly. All going well, the therapy could be launched at the end of 2016, or early 2017.
Ionis licensed and developed the drug, as part of its pipeline of 38 antisense therapies. Boyce said the company is now in the process of transferring ownership to Biogen, with whom they have a close and strategic partnership.
The drug has been studied in both presymptomatic and symptomatic patients covering Types 1, 2, and 3. All Types are caused by the same SMN1 mutation, but differ based on the age of onset and the severity of the disease.
Boyce said it was too early to comment on pricing and that Biogen will be making the decision when the time comes.
All SMA types can be detected with carrier screening, but insurers typically offer reimbursement only when the patient has a family history of the disease.
That’s consistent with current guidelines from the American College of Obstetricians and Gynecologists’ (ACOG) Committee on Genetics, which state that “preconception and prenatal screening for SMA is not recommended in the general population at this time.”
That could change in the coming years as diagnostics companies deploy next-generation sequencing panels that can test for hundreds of different disorders.
Photo: BlackJack3D, Getty Images
