Dive Brief:
- Roivant subsidiary Enzyvant this week announced that it is will begin a rolling Biologics License Application for its lead compound RVT-802. The company hopes to complete the application by the end of the year.
- The drug, which will be the first Roivant compound to face regulators, is designed to treat a rare pediatric disease called complete DiGeorge Anomaly, which affects about 10 to 20 infants in the U.S. each year.
- The compound is a regenerative tissue therapy produced from donated thymus tissue. The therapy was developed under a collaboration with Duke University.
Dive Insight:
Roivant Founder Vivek Ramaswamy is bullish on his accelerator potentially bringing its first product to the market, setting up expectations that RVT-802 could reach markets quickly.
Enzyvant is already talking with payers across both the public and private space to develop a value-based model for rolling the drug out. The company believes what it comes up with could be a blueprint for other, more complex value-based pacts in the future.
"It's about as black and white as you can get in regards to the outcomes," said Enzyvant CEO Alvin Shih during a Roivant pipeline day on Tuesday afternoon. "We also have a clearly defined and identifiable patient population that is not overly large, there is also a clear unmet medical need, and we know that the distribution model will be relatively simple given the closed supply chain," he added.
Both executives believe the treatment is the perfect candidate for a value-based pricing model because the outcome is incredibly (if morbidly) easy to measure — either the patient lives after getting treatment or they die.
RVT-802 is a one-time therapy that uses donated thymus tissue usually left over after infants have cardiac surgery. Shih told BioPharma Dive that supply is not a problem because there is usually an overabundance of this tissue that goes unused. Each donation typically results in one dose of the drug, but a small patient population means there potentially only needs to be about 10 to 20 doses made each year.
The processing and culturing done to the tissue to turn it into RVT-802 was designed so that recipients don't reject the donated tissue.
Since the therapy was developed at Duke, the two organizations have years of patient data — with the oldest patient to get the therapy recently turning 25 years old, said Shih. Patients with the disease have a low T-cell count and are unable to develop a fully formed immune system. Most children born with the condition typically die within the first 24 months.
But the company isn't leaving anything to chance: Enzyvant hired Morgan Molloy as its chief commercial officer. Molloy spent several years launching rare disease products for Sanofi Genzyme, and more recently spearheaded the commercialization team at Spark Therapeutics, where he worked to develop the value-based contract plan the biotech recently set up for its one-time gene therapy.
Molloy admitted that the plans for Spark's Luxturna were much more complex because of the harder-to-measure outcomes, but believes the industry is going to continue seeing more and more drugs trying to bring these types of pricing models forward.
Enzyvant is one of twelve "Vants," or subsidiaries, under the Roivant umbrella. But unlike the typical holding company, Roivant acts like more of an accelerator, handling the business development and some of the SG&A and legal work, but allowing each Vant to function autonomously, with its own management and unique therapeutic area.
While the model has its critics and has met its fair share of setbacks, Ramaswamy has stood out as an innovator in the field. He told BioPharma Dive at the event that he doesn't expect the model to be accepted outright by traditional pharma, but hopes to prove that it can work.