Despite the numerous challenges of bringing a new rare disease treatment through development to approval, the drug pipeline for spinal muscular atrophy (SMA), a neuromuscular disease that is the leading genetic cause of death for children under age two, has continued to grow aggressively. Read more about how Cure SMA is working hard to continue this forward momentum.
In a 2014 study, Tufts University estimated that it can take upwards of $2.6 billion to bring a drug through development to FDA approval. That number is daunting in any case, but particularly for rare diseases. Furthermore, rare disease populations are often diverse, dispersed, or both, making drug development even more complex.
Despite these challenges, the drug pipeline for spinal muscular atrophy (SMA), a neuromuscular disease that is the leading genetic cause of death for children under age two, has continued to grow aggressively. We began tracking the SMA drug pipeline in 2000, when there were just two early-stage programs. We now have 18 active programs spread among multiple approaches, with seven programs in clinical trials, and 16 pharmaceutical companies investing in SMA drug development.
Our organization, Cure SMA, has provided funding for half of the programs currently in development. In total, we’ve invested $59 million through more than 200 research grants given over the course of the last 30 years. That volume and longevity has been crucial in advancing the SMA drug pipeline. But our most powerful tool is our aggressive yet strategic research model.
In order to maximize return, we invest in four different stages of research—basic research, drug discovery, clinical trials, and clinical care—and in four different possible therapeutic targets. Like any investor with a diverse portfolio, we balance risk through this strategy.
We also place emphasis on “seed funding” for early-stage research projects, to prove their worth as possible avenues to drug discovery. In doing so, we lower the risk and attract larger investments from industry, government, and other funding sources as drug candidates move toward potential FDA approval.
A second major prong of our research strategy is based on our close connections to the patient community. These connections allow us to provide drug developers with access to patient data, assistance with clinical trial recruitment, advocates for regulatory decisions, and more.
Our family support programs—such as our information packets, care packages, equipment pool, and Annual SMA Conference—first and foremost provide families with assistance and hope. However, they also provide incredible incentives to join together and build a strong and unified community. We are then able to offer our community as an extraordinary resource for drug developers.
Though the drug pipeline continues to grow, no SMA therapy has yet come forward for FDA approval. As we approach this next challenge, we’re undertaking new initiatives to broaden our community even further, building stronger relationships with our government and regulatory partners in addition to our pharmaceutical industry collaborators. When the time comes for the FDA to consider an SMA drug for approval, we want to be sure our voice is heard. The same strategic, community-based model that has gotten us this far will take us to our ultimate goal: an FDA-approved treatment for SMA.