Go or no go? Merck, Regeneron and Alexion await US expansion

Approvals for new uses of old drugs should far outweigh nods for novel projects in June.

Has the summer lull begun early? Only a handful of novel projects are set to come before the US FDA next month, including Xeris’s glucagon pen for severe hypoglycaemia, and Amag and Palatin’s Vyleesi for female sexual dysfunction.

But the agency will be kept busy with decisions on new indications for big products like Merck & Co’s checkpoint inhibitor Keytruda, Sanofi and Regeneron’s Dupixent, and Alexion’s rare disease blockbuster Soliris.


The FDA already got a head start on its June to-do list this week, giving an early thumbs-up to Celgene’s Revlimid in previously treated follicular lymphoma or marginal zone lymphoma; a decision had been due by June 27.

Also approved well ahead of their June PDUFA dates were Inlyta combinations with Merck & Co’s Keytruda and Pfizer and Merck KGaA’s Bavencio in first-line renal cancer, setting up a checkpoint inhibitor battle in this new indication; both will be challenging Bristol-Myers Squibb’s Opdivo, approved for this use last year.

Renal cancer is not Keytruda’s only potential new money-spinner: the FDA is also due to rule next month on its approvability in first-line head and neck cancer and third-line small-cell lung cancer. However, it is unclear how much of an impact the PD-1 inhibitor can make in these settings (Keytruda looks to secure a June double play, May 28, 2019). 

Merck is also due a decision on a new use for the antibiotic combo Zerbaxa, but this is hardly a big product for the company: total sales of $256m are forecast in 2024, according to EvaluatePharma consensus.

Meanwhile, Dova could get a new lease of life if Doptelet can expand into chronic immune thrombocytopenia. The platelet booster is already approved for thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a medical procedure, but the launch has progressed slowly, with questions around the drug’s price and competition.

Other supplemental approvals slated for June involve Sanofi and Regeneron’s Dupixent in nasal polyps, and Alexion’s Soliris in neuromyelitis optica spectrum disorder. The new indication for the latter could add sales of $350m by 2024, the sellside believes, and approval looks like a dead cert after impressive data from the Prevent trial.

Supplementary and other notable approval decisions due in June
Product  Company Event type Date
Zerbaxa Merck & Co sNDA for adult nosocomial pneumonia Jun 3
Keytruda Merck & Co sBLA for first-line head and neck cancer  Jun 10
Keytruda Merck & Co sBLA for third-line small-cell lung cancer Jun 17
Dupixent Regeneron/Sanofi sBLA for chronic rhinosinusitis with nasal polyposis Jun 26
Soliris Alexion Pharmaceuticals sBLA for neuromyelitis optica spectrum disorder Jun 28
Doptelet Dova Pharmaceuticals sNDA for chronic immune thrombocytopenia Jun 30
Thiola Retrophin sNDA on new formulation of Thiola in cystinuria Jun 30
Source: EvaluatePharma.

The outlook is more uncertain for Amag and Palatin’s subcutaneous female sexual dysfunction candidate Vyleesi, which was originally due an FDA decision in March, only to have the date put back to June after the agency requested phase I data on ambulatory blood pressure.

Amag has stressed that Vyleesi does not have any known alcohol interactions, a side effect that scuppered Sprout Pharmaceuticals’ – now Bausch Health Companies’ – Addyi.

Another first-time approval decision set for June involves Xeris Pharmaceuticals’ lead project, the Gvoke Hypopen, a glucagon autoinjector designed as an emergency treatment for diabetics suffering from hypoglycaemia. The company is also investigating glucagon pens for other uses, including exercise-induced hypoglycaemia and congenital hyperinsulinism, so the FDA verdict will be a key test of its approach.

Finally, Acer Therapeutics will find out whether it can market Edsivo for vascular Ehlers-Danlos syndrome, a rare disorder of collagen production.

Notable first-time US approval decisions due in June
Project Company PDUFA date Product NPV ($m)
Gvoke HypoPen (G-Pen) Xeris Pharmaceuticals Jun 10 713
Vyleesi (Rekynda) Amag/Palatin Technologies Jun 21 507
Edsivo Acer Therapeutics Jun 25 122
Source: EvaluatePharma.

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