Orphan drug designation grants financial incentives to companies developing medications for rare diseases, which might not be profitable enough to pursue without government assistance.
While a Phase 1 clinical trial investigating Prosetin was due to start this summer, the COVID-19 pandemic delayed previously ongoing preclinical and clinical research. However, Project ALS’ goal is to initiate human clinical studies by the end of this year.
Developed by Project ALS at Columbia University, Prosetin reverses cells’ endoplasmic reticulum stress (a kind of stress response to defective protein production) that causes a buildup of toxic compounds within nerve cells. It does this by blocking the action of a family of proteins called MAP4 kinases (MAP4K).
Using an ALS mouse model, Project ALS researchers showed that orally administered Prosetin crossed the blood-brain barrier (a semipermeable membrane that protects the brain and spinal cord) where its potent inhibition of MAP4K proteins and anti-inflammatory effect improved the survival of motor neurons.
In a press release, Project ALS notes that the Orphan Drug Act (ODA) of 1983 led to a “sea change” in the development of therapies for rare disorders, which it defines as those affecting fewer than 200,000 Americans.
While the FDA approved only 10 rare disease products during the 10 years prior to the ODA’s passage, it has approved more than 450 new rare disease products since then. In 2017, 39% of all new medicines that the FDA approved were orphan drugs.
The handful of representative diseases first named in the ODA included ALS, making potential treatments for that condition strong candidates for orphan status.
While Prosetin’s orphan status will not change the timing or viability of clinical trials, it does make it eligible for research grants specific to orphan drugs. These could support more rapid and robust efforts to develop Prosetin as a clinically useful compound. It also allows Prosetin’s developers to seek additional FDA help and guidance when designing clinical trials.
Should Prosetin show promise during clinical development, its orphan status will bring more incentives to Project ALS and any future partners, helping them accelerate Prosetin to market.
Although the incentives and special oversight that accompany orphan status provide ways to accelerate a potential therapy’s development, that therapy will still be held to the same standards of safety and effectiveness as any other FDA-regulated investigational compound.
With Prosetin, Project ALS aims to be the first ALS non-profit organization to sponsor an investigational new therapy for the treatment of that disorder.
“We are committed to transparent updates every step of the way,” Project ALS states in its press release, “… and remain focused on our goal of getting Prosetin to clinical trial as an FDA-defined investigational new drug as efficiently and responsibly as possible.”
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