Optum spotlights orphan drugs in drug pipeline report

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Optum is seeing an increase of new orphan drugs, and expects this trend to continue, if not accelerate, according to a drug pipeline report from the UnitedHealth Group subsidiary.

What makes this important, said Sumit Dytta, chief medical officer at OptumRx, is that orphan drugs are priced more than 30 times higher than non-orphan drugs. Almost 40% of new orphan drugs coming to market cost more than $100,000 per year, while cell and gene therapies can cost much more.

One of the orphan drugs in the pipeline, Sparsentan, is currently under review by the Food and Drug Administration and may be approved by the end of the year. It has the potential to treat immunoglobulin A (IgA) nephropathy, also known as Berger's disease.

IgA is an immune system antibody that attacks pathogens and fights infections. In IgA nephropathy, this antibody collects in the kidneys, causing inflammation and gradual damage to kidney tissues. Over time, IgA nephropathy can lead to end-stage kidney disease (ESKD) and the need for dialysis. IgA nephropathy is one of the most common kidney diseases, affecting more than 100,000 people in the U.S. Travere Therapeutics, the drug's manufacturer, estimates about 30,000 to 50,000 cases are addressable with sparsentan at launch.

There is an unmet need for effective IgA nephropathy treatments, said Optum. Sparsentan would potentially be the first non-steroidal drug approved for treatment of IgA nephropathy.

WHAT'S THE IMPACT?

Optum also highlighted Etranacogene dezaparvovec, a new gene therapy with orphan status for hemophilia B. It will be the first gene therapy approved for hemophilia B administered as a one-time infusion directly into the bloodstream.

Hemophilia is an inherited bleeding disorder primarily affecting males in which the blood does not clot properly. There are two major types of hemophilia: A and B. People with hemophilia B have low levels of the clotting factor IX (FIX). Current treatment includes replacement therapy to supply missing clotting factor from an external source.

FIX replacement therapy has a high treatment burden and is extremely costly, said Optum. A 2021 study analyzed patients with severe and moderately-severe hemophilia B. Lifetime FIX replacement costs ranged $21 to $23 million dollars, depending on the type of treatment.

If approved, etranacogene dezaparvovec would be the first one-time gene therapy for hemophilia B and reduce, and in some cases eliminate, the need for chronic and as-needed FIX replacement therapy. But of the estimated 6,000 hemophilia B cases in the U.S., the manufacturer is targeting moderate-to-severe hemophilia B patients, representing approximately 60% of all patients – thus narrowing the market and straining market sustainability.

The third and last drug reviewed by Optum, Pegcetacoplan, will treat geographic atrophy, a late-stage form of an eye condition known as age-related macular degeneration. Recently accepted for FDA review, a decision is expected before the end of November.

Geographic atrophy is an aggressive form of dry (as opposed to wet) age-related macular degeneration (AMD). Geographic atrophy is a leading cause of blindness that affects more than one million people in the U.S.

There is no FDA-approved treatment for geographic atrophy, and existing treatments for wet AMD have not been effective for geographic atrophy. Over the next few years, this is likely to change significantly, according to Optum. In addition to pegcetacoplan from Apellis Pharmaceuticals, at least 10 pharmaceutical companies have products for geographic atrophy in development.

THE LARGER TREND

Just last week, President Biden issued an executive order detailing new payment and delivery models intended to lower the prices of prescription drugs. The order focuses on models that can be tested under the Center for Medicare and Medicaid Innovation.

The order follows August's passage of the Inflation Reduction Act of 2022, which was intended to protect beneficiaries from high drug costs by phasing in a cap for out-of-pocket costs at the pharmacy, and establishing a $35 monthly cap per prescription for insulin covered by a Medicare prescription drug plan, as well as insulin delivered through traditional pumps.

Per the order, the secretary for the Department of Health and Human Services will be able to negotiate prices for selected high-cost prescription drugs for Medicare beneficiaries.

HHS' analyses of prescription drug prices from 2016-2022 show that if the Inflation Reduction Act had been in place from July 2021 to July 2022 more than 1,200 prescription drugs potentially would have been subject to the new provision requiring drug manufacturers to pay rebates to Medicare if they enact price increases greater than inflation for drugs. Price increases on those drugs in the month the price change took effect averaged more than 30%.

Twitter: @JELagasse
Email the writer: jeff.lagasse@himssmedia.com