When seeking to take a product to market in the U.S., it helps to have a clear idea of just what that product is. OvaScience (OVAS) felt that Augment, a technique for improving the chances of successful IVF, fell into the FDA’s category for tissue-based products. The FDA felt otherwise.
The agency has demanded that the company file an IND before continuing with its first human trial, thereby condemning it to a much longer path to market which it will struggle to afford. More broadly, the regulatory situation across the board for IVF technologies and beyond could change if the FDA starts regulating cell-based technologies as drugs.
OvaScience had informed the FDA that it believed that Augment came under the agency’s human cellular and tissue-based products (HCT/Ps) regulations when it started the first human trial at the beginning of this year. Such products do not require regulatory approval for testing or marketing.
But, somewhat precipitately, it then pushed forward with the study without waiting for the FDA’s reply. The reply, a so-called “untitled” letter saying that Augment would indeed need an IND and would have to be approved under a biologics license application (BLA), came in the last few days.
At issue is the degree to which OvaScience’s technology alters human egg cells. The current regulations state that a product may be regulated under the HCT/P rules if it is “only minimally manipulated” – that is, if its processing does not alter “the relevant biological characteristics of the cells or tissue”.
Augment involves removing the mitochondria from a woman's egg precursor cells and injecting them into one of her egg cells, along with a sperm cell, during IVF. Adding the mitochondria is intended to permit women with poor-quality gametes – those damaged by chemotherapy for instance – to conceive with their own eggs rather than donated ones. It could also eliminate the need for the hormonal hyperstimulation usually required in IVF.
The FDA is unconvinced that this counts as minimal manipulation. OvaScience hopes that it might be open to persuasion, though, and says it will meet the agency to try to get it to drop the IND requirement.
The hope for OvaScience now lies away from the U.S. The firm contends that the international IVF market is 10 times larger than that in the U.S., and while this is perhaps an overstatement it is true that the US is less crucial for IVF than it is for medical technologies in general (EP Vantage interview – Auxogyn’s viability boosted by embryo selection tool, July 15, 2013).
Be that as it may, Augment is now stalled in the U.S., where its clinical trial has had to be halted; the setback was significant enough to cause OvaScience’s share price to drop 38% yesterday. Leerink Swann analysts give Augment a 30% chance of launching in the U.S., if an FDA approval is not required.
OvaScience does not have the cash to fund the kind of studies mandated by an IND. The company therefore has two options if it is to keep going: either it gets Augment on the European and Asian markets very fast indeed and ramps the kind of sales that enable it to conduct IND studies in the US or it twists the FDA’s arm and goes down the HCT/P path after all.
Augment is the company’s only clinical-stage product. If it cannot make either of these options work its future looks bleak. It is apparently unwise to proceed with development without first checking that the FDA is happy.