Two major changes over the past year indicate that the FDA is preparing to substantially lower the requirements for approval of stem cell therapy and other regenerative medicine therapies.
First, the 21st Century Cures Act, passed last year, contains a provision supporting an accelerated pathway for regenerative medicine.
Second, Trump's new FDA commissioner, Scott Gottlieb, has consistently favored lowering the bar for drug and device approval, and it is now clear that this includes stem cell therapy. Last week he outlined in a speech the "steps FDA is taking to modernize its regulatory approach to new cell and gene based therapies".
At least two companies are already seeking to take full advantage of these lowered requirements.
One company, Mesoblast, recently announced plans to exploit "a potential accelerated pathway for its proprietary allogeneic mesenchymal precursor cell (MPC) product candidate (MPC-150-IM) for patients in the most advanced stages of chronic heart failure, defined as New York Heart Association (NYHA) stages Class III and Class IV."
According to the company: "Based on cumulative clinical results to date and the serious and life-threatening nature of this disease, we believe there is a pathway for accelerated entry of this product candidate into the market to provide a paradigm shift in treatment."
The company is performing two clinical trials, a phase 2b trial and a phase 3 trial. The phase 2b trial includes 159 patients with end-stage heart failure receiving support from a LVAD. The primary endpoint of the trial is the amount of time patients can be weaned from the LVAD in the 6 month period after randomization. The ongoing phase 3 trial will randomize 600 patients with mild to moderate CHF to active treatment or sham-treatment. The company belies that a positive result in the phase 2b trial, which it expects to complete in the first half of next year, along with positive interim results of the phase 3 trial, could serve as the basis for FDA approval.
Another company that will likely seek to take advantage of this pathway is Capricor, the stem cell company founded by the prominent Cedars-Sinai researcher Eduardo Marbán. I've written previously about the company's efforts to spin negative results. A recent press releasefrom Cedars-Sinai for a study in ratsbegins: "Cardiac stem cell infusions could someday help reverse the aging process in the human heart, making older ones behave younger, according to a new study from the Cedars-Sinai Heart Institute." Tim Henry, a Cedars-Sinai cardiologist who is a co-principal investigator of the Capricor trials, confirms that the company hopes to use the latest data from its clinical trials to support an early approval. But the company's main hopes now focus on an extremely narrow niche indication for hypertrophy associated with Duchenne muscular dystrophy (DMD). Capricor appears to be following the controversial model successfully adopted by Sarepta, which gained approval for a DMD drug with only the scantest evidence of efficacy.
Capricor CAPR on NASDAQ has risen from $1.00 to $2.46 over the last month.
Market Cap Approx US$60m. Young Company.
http://capricor.com/product-pipeline/
Mesoblast MSB has risen from $1.40 -$1.88 over the last month.
Market Cap Approx AU$880m. Mature Company.
http://www.mesoblast.com/product-candidates/product-candidates-overview
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