Planned 31 May revocation of enforcement discretion for certain cell therapy products without IND or BLA is important, but experts say the FDA’s next moves are what will really shape the field and patient safety moving forward.
The Alliance for Regenerative Medicine (ARM), a trade association representing regenerative medicine firms, applauded Marks’ words and the end of the discretionary enforcement era.
The US Food and Drug Administration has approved only 7 cell and gene therapy drugs, but the new product pipeline is teeming with approximately 1,200 experimental therapies, more than half in Phase 2 clinical trials. And the number of cell and gene therapy developers is rising rapidly; the Alliance for Regenerative Medicine estimates 1,100 such ventures in 2020, an increase of about 10% over 2019.
Coronavirus or not, the progress of advanced medicines like gene and cell therapies steamed ahead in 2020, according to the latest annual report out from the Alliance for Regenerative Medicine today.
Record levels of funding are flowing into cell and gene therapy companies like Jaguar and Taysha. Last year, nearly $20 billion was invested in companies in the sector, more than 50% higher than the previous record set in 2018, according to numbers compiled by the Alliance for Regenerative Medicine.
With less uncertainty surrounding the pandemic, the biopharma industry appears on track to shift efforts back to its internal programs and possibly meet, if not exceed, the record-breaking deal level of 2020. Strong financial markets, however, may continue to hold M&As down.
The Alliance for Regenerative Medicine argued that the MACPAC recommendation would hurt “vulnerable” patient populations disproportionately and deter investment over the longer term.
More regulatory harmonization in the ultra-rare disease space is desirable and would make treatments for these conditions more commercially viable, said regulators at the online Cell and Gene Meeting on The Mediterranean, organized by the Alliance for Regenerative Medicine.
