Pharma, R&D

The top 10 orphan drugs in the late-stage pipeline

Orphan drugs have come into the spotlight, scaring up headlines involving big prices and small patient populations. Some companies have been accused of profiteering by manipulating the FDA’s rules on this — hello, Marathon — but orphan therapies remain a very closely watched arena with some major new prospects coming through the late-stage pipeline. And some of these drugs are offering patients real hope for the first time.

Every year, Evaluate crunches the numbers on the top prospects in the pipeline, and the company’s editorial arm EP Vantage includes it in their annual report on orphan drugs. I’ve picked up their list and rearranged it by Evaluate’s sales estimates for 2022.

A word of caution. Sales estimates are easy to make and hard to deliver on, so it’s always important to take these numbers with a grain of salt. Also, some of these drugs still face big questions as they come under FDA review. And the FDA review process itself may well change radically, especially for orphan drugs, as President Trump demands faster response times on this front.

It’s still early to be counting chickens, but these eggs deserve careful attention.

Tesaro: niraparib, PARP/cancer
2022 sales: $1.8B

Two years made a big difference for Tesaro $TSRO. In 2015, Evaluate forecasted this drug would bring in $499 million in 2020. Now under review as the biotech explores a broad label, analysts have been hiking their projections, which keeps this company on most analysts’ lists of likely takeover targets. Ovarian cancer qualified this drug for orphan status.

Kite Pharma: axiabtagene ciloleucel, CAR-T/cancer
2022 sales: $1.7B

Kite’s new name for KTE-C19, its top CAR-T in the pipeline, is a tongue twister, but after the biotech unveiled 6-month data on Tuesday, we should all learn how to pronounce it. The first clear evidence of durability leaves Kite $KITE in an impressive position as it looks to beat out Novartis with the first generation of CAR-Ts. It should be quite a rivalry.

Roche: emicizumab (ACE910)/ hemophilia
2022 sales: $1.5B

Maybe we shouldn’t get too far ahead of ourselves on this one. Yes, Roche $RHHBY has very high expectations for this drug. But it’s also been plagued by safety questions, which have yet to be fully aired by regulators. A top prospect? You bet. But don’t count on the money yet.

AstraZeneca: tremelimumab/CTLA-4 checkpoint inhibitor
2022 sales:  $1.3B

Usually mentioned right alongside durvalumab, AstraZeneca’s $AZN other checkpoint, the tremelimumab combo emerges as an absolutely central feature in distinguishing the pharma giant as it tries to leapfrog some very advanced players like Merck, Bristol-Myers and Squibb. This one is do or die for AstraZeneca, which won orphan status for malignant mesothelioma.

Shire: lanadelumab (SHP643)/hereditary angioedema 
2022 sales:  $1.1B

This was the jewel in the crown for Shire {SHPG} when it paid $5.9 billion for Dyax back in late 2015. The drug blocks enzyme plasma kallikrein to treat hereditary angioedema, and Shire execs have already indicated that they believe it can reach $2 billion in annual sales. And last summer this drug figured prominently among the top three programs featured by CEO Flemming Ornskov. It has a breakthrough drug designation and a shot at glory.

Teva: SD-809/tardive dyskinesia
2022 sales:  $1B

Teva $TEVA already has had to deal with a serious setback on this drug when the FDA rejected its application for Huntington’s chorea last year. But it’s still shooting for a blockbuster with a new drive on to get this approved this year for tardive dyskinesia. A seemingly permanently troubled company, Teva needs this win badly. Teva’s big program for this drug puts it in a head-to-head showdown with Neurocrine $NBIX, which noted recently that it filed for an approval on the rival drug valbenazine. And some analysts would put Neurocrine’s drug well ahead of Teva’s on this one.

bluebird bio: LentiGlobin/gene therapy
2022 sales:  $969M

Bluebird $BLUE helped make gene therapy a hot field again, and its struggles with varying degrees of impact on patients has helped raise fresh questions about where it’s headed on beta thalassemia, where investigators are now pursuing a Phase III trial. Potential? You bet. But there are still plenty of unanswered questions.

AstraZeneca: acalabrutinib (ACP-196) /BTK inhibitor 
2022 sales:  $968M

AstraZeneca won’t mind being the only company to make two appearances on this top 10 list. This drug came its way when it acquired a majority interest in Acerta in 2015, as Pascal Soriot sought to execute a major turnaround in the pharma giant’s prospects. With generic competition eating away at revenue, AstraZeneca needs all the help it can get from its oncology pipeline. This drug is in late-stage studies for B-cell blood cancers.

Novartis: CTL019/CAR-T
2022 sales: $917M

This is the “other” CAR-T you probably heard about. After Novartis ripped up its cell therapy unit last year, the pharma giant went to some trouble to try and reassure observers that it was still on track to file for an approval in early 2017. But analysts are discounting its impact compared to Kite, which remains the leader in the field, regardless of who gets to the FDA first.

AbbVie: veliparib/PARP inhibitor
2022 sales: $854M

PARPs are all the rage these days, especially since Pfizer picked up talazoparib in its $14 billion Medivation buyout. AbbVie, though, remains well behind what is becoming a fairly crowded field. That leaves them on the list for now, but just barely, as all eyes remain on Tesaro.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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