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In our constant endeavor to find companies ready to launch products and services in the gene therapy space, we come across Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP), who alongside bluebird bio, are penning a new chapter in the story of gene therapy for sickle cell disease.

Now, to bring this into context, the recent report by the Institute for Clinical and Economic Review (ICER) has illuminated our understanding of exagamglogene autotemcel (exa-cel) from Vertex/CRISPR and lovotibeglogene autotemcel (lovo-cel) by bluebird. For those unacquainted with ICER, it’s more than just a research body. Its autonomous nature and dedication to understanding clinical and economic merits make its insights invaluable, especially in the convoluted landscape of medical pricing and accessibility in the US.

Though ICER doesn’t wield regulatory might, its voice is influential, echoing in the hallways where policies are crafted and pricing decisions are made. In a world where pricing and accessibility are continually debated, having such non-partisan guidance is a boon.

However, the imminent FDA verdict in December has not yet drawn a lot of stock price action.

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Source: Seeking Alpha

Beyond the raw data, a narrative is unfolding. Independent committees have lent weight to the potential of both therapies, suggesting they might eclipse current standards. And in a bold and forward-looking move, ICER is nudging manufacturers and payers to navigate uncharted waters – devising novel payment structures that balance immediate financial realities with the enduring promise of gene therapy.

Diving into the numbers, certain assumptions offer a glimpse into potential financial futures. Consider a shared patient pool for Exa-cel and Lovo-cel, with a treatment price tag of $2 million. Under this light, Exa-cel’s annual revenue could be envisioned at a little less than $400 million, assuming the Lovo-cel will rake a similar amount. Factor in uncertainties in costs, and operating profits could hover around $180 million in the early phase for each program. But as any seasoned investor knows, numbers are a blend of facts, estimations, and the unpredictable dance of market forces.

But, in the spirit of long-term insight, these developments, despite their intricacies, are heartening.

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