How will emerging SMA therapies address longstanding unmet needs, and where will remaining market opportunies lie?

Key findings: Biogen/Ionis’s disease-modifying antisense oligonucleotide therapy Spinraza has revolutionized the treatment of spinal muscular atrophy (SMA). Recent evidence indicates that SMA patients who receive Spinraza, on average, live longer and achieve motor milestones that would not be typically expected of patients with the natural history of their disease subtype (1,2). On the heels of the transformative clinical benefits of Spinraza treatment in type I and II SMA patients, several new therapies are poised to bring more conveniently-delivered options, broader tissue distribution than Spinraza, and a potentially curative single-administration gene therapy. Furthermore, interviewed experts note that early and, eventually, presymptomatic diagnosis and treatment of SMA will be critical to maximize the clinical benefit of treatment to patients; indeed, developers of current and emerging agents are actively assessing the efficacy, safety, and tolerability of their assets in the genetically-defined presymptomatic population.

The “so what” for decision makers:

Spinraza has revolutionized SMA care by providing patients with a lifesaving therapy that acts directly on a genetic modifier of SMA severity. However, the agent has faced payer pushback over its high cost and it requires repeated, burdensome intrathecal delivery—key challenges for patients, caretakers, and treatment providers. Thus, clinical and commercial opportunity remains in this arena.

Although the SMA pipeline is relatively small, DRG forecasts the launches of a symptomatic therapy and three new disease-modifying therapies (DMTs) by 2027; like Spinraza, emerging DMTs are targeting the underlying genetic defect in SMA. Launches of the DMTs, which comprise a corrective gene therapy encoding SMN1 (Novartis/AveXis’s AVXS-101) and two oral SMN2 splicing modulators (Roche/PTC/Chugai’s risdiplam and Novartis’s branaplam), will begin 2019, providing patients with greatly-needed additional treatment options. The DMTs are poised to compete for market share; however, the curative potential and single IV administration of AVXS-101, as well as its projected launch ahead of the two oral SMN2 splicing modulators, will cement its position as market share leader in SMA by 2027.

Key questions answered in the analysis:

  • How large is the treatable SMA population and how will its size change between 2017 and 2027?
  • What is the current state of treatment in SMA? How many patients within each disease subtype are receiving Spinraza?
  • What clinical needs remain unfulfilled?
  • What pipeline products are viewed by SMA treatment providers as most promising, and what sales/uptake will they achieve in SMA? Which notable early-stage therapies are progressing through development?
  • What are the drivers and constraints in the SMA market, and how will the market evolve over the forecast period?

Markets covered: United States, France, Germany, Italy, Spain, and United Kingdom.

Methodology: Six country-specific interviews with thought-leading neurologists supported by survey data from fifty SMA treatment providers.

Key drugs covered: Spinraza (nusinersen), AVXS-101, risdiplam, branaplam, reldesemtiv

Key companies mentioned:  Biogen, Ionis Pharmaceuticals, Roche, PTC Therapeutics, Novartis, Cytokinetics, Astellas Pharma.


  • Finkel RS, et al. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017;377(18):1723-1732
  • Mercuri E, et al. Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy. N Engl J Med. 2018;378(7):625-635.

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