BURLINGTON, Mass., Aug. 12, 2015 /PRNewswire/ -- Decision Resources Group forecasts that the number of lysosomal storage disorders (LSDs) with available treatments is set to double from eight in the base year of its forecast (2013) to sixteen by 2023. As a consequence, the value of the LSDs market is set to increase from $3.2 bn in 2013 to $5.9 bn in 2023. Additionally, the development of therapies with novel mechanisms of action will provide competition for traditional enzyme replacement therapy (ERT) for a number of LSDs.
Other key findings from the Pharmacor report entitled Lysosomal Storage Disorders:
- Small molecule therapies have potential to significantly reduce the dosing burden for patients with LSDs: The emergence of oral therapies (e.g. substrate reduction therapies [SRTs] such as Cerdelga [Sanofi's eliglustat], and chaperone therapies such as Galafold [Amicus Therapeutics' migalastat]) offers significant dosing advantages over currently used intravenously-administered ERTs for the treatment of Gaucher's disease and Fabry disease, respectively. Lack of long-term outcomes data and physician satisfaction with current treatments may limit the use of these novel agents, at least in the short-term. Despite this, both therapies are forecast to capture a significant portion of the market for their respective indications.
- The launch of first-ever treatments for a number of LSDs will significantly improve patient outcomes for afflicted patients: The anticipated availability of therapies for an additional eight LSDs will significantly improve the long-term prospects for patients with previously untreated diseases. Patients with Morquio syndrome A and lysosomal acid lipase (LAL) deficiency are first in line to benefit from the emergence of novel treatments, with the respective launches of Vimizim (BioMarin's elosulfase alfa) and Kanuma (Alexion/Synageva's sebelipase alfa) in 2014 and 2015 respectively.
- Therapies that can address the central nervous system (CNS) complications of LSDs are expected to launch for the first time: LSDs are characterized by deficiencies in various enzymes, that in more severely afflicted patients result in considerable neurological impairment. Standard ERTs are unable to cross the blood-brain barrier (BBB), meaning that LSDs with a CNS complication have previously been untreated. However, a number of companies are developing a range of ERTs for intrathecal or intracerebroventricular administration. These therapies are expected to launch during the forecast period, expanding treatment coverage to the more severely afflicted patients.
- Significant opportunities exist for drug developers that can identify potential therapies for LSDs: Despite the anticipated expansion of LSDs with treatments from eight to sixteen during the forecast period, over thirty LSDs remain without a current treatment or a late-stage candidate in development. Therapies for LSDs typically receive breakthrough orphan drug designation allowing for accelerated regulatory review. Additionally, due to the limited patient populations for individual LSDs, therapies for these diseases command extremely high prices, and receive nearly universal payer reimbursement. As such, development of therapies for LSDs represents a significant opportunity for drug developers.
Comments from Decision Resources Group Senior Business Insights Analyst Eamonn O'Connor, Ph.D.:
- "The landscape for Lysosomal Storage Disorders is due to undergo a profound change during the forecast period. In addition to the expansion in the number of LSDs with therapeutic coverage, the emergence of treatments with novel mechanisms of action, and novel dosing formulations, will allow for a significant increase in LSD patients receiving treatment. This will have the dual effect of significantly improving these patients' quality of life, and also significantly increasing the value of the LSD market."
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SOURCE Decision Resources Group