Go or no go? Novartis, Sanofi and Regeneron await US verdicts

May looks to be a fairly quiet month for US drug approvals, though Novartis's gene therapy should make headlines.

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Next month might not boast a long list of impending FDA approvals, but it could well yield one of 2019’s most significant new arrivals: Novartis’s gene therapy Zolgensma, for the childhood wasting disease spinal muscular atrophy.

Vantage’s monthly look at the FDA’s calendar also reveals a first-time US decision for Sanofi’s controversial malaria vaccine Dengvaxia, and a handful of notable supplementary approvals, including a potentially useful new indication for Regeneron’s eye drug Eylea. Meanwhile, Pfizer and Merck KGaA will be wondering whether Bavencio might get an early nod for expanded use in first-line renal cell carcinoma, after Keytruda won a super speedy green light in the same setting this week. 

Notable first-time US approval decisions due in May
Product  Company PDUFA date  Product NPV ($bn)
Dengvaxia Sanofi May 1 n/a
Slinda Insud Pharma May 27 n/a
Zolgensma Novartis Early May 7.9
Source: EvaluatePharma. 

Opening the month will be a decision on Sanofi’s Dengvaxia, which is already used in many parts of the world but has yet to garner a US marketing licence. Unforeseen safety issues have blighted this product in countries like the Philippines, making the drug’s future hard to call. An FDA advisory committee recommended approval last month, though not unanimously, while a lack of other options could perhaps works in Dengvaxia’s favour (More Sanofi dengue setbacks could benefit Takeda, March 8, 2019). 

The exact PDUFA date for Novartis's Zolgensma is unknown, but the company has said early May and, given the dramatic results that the gene therapy has generated, an approval could well come early. Alongside quarterly results today executives insisted that the regulatory review remained on track; earlier this week Novartis revealed that two infants in two separate trials had died. One case has been confirmed as unrelated to the therapy, though the other has yet to be explained.

Still, spinal muscular atrophy is frequently fatal so a green light is widely expected. The final price is probably the biggest unknown here, with many expecting Zolgensma to emerge as the world’s most expensive drug.

Elsewhere, the private Spanish group Insud Pharma should hear by the end of the month whether its progesterone-only contraceptive pill has passed FDA muster. This contains the synthetic progestin drospirenone and is trade-marked Slinda, and the company claims that the pill could carry lower cardiovascular risks than combined hormonal contraceptives.

While drospirenone is available in combined products like Bayer’s Yaz, it has yet to be approved as a single agent; Insud claims that its 24/4 dose regimen has demonstrated convincing efficacy as a contraceptive. 

Supplementary and other notable approval decisions due in May
Product  Company Headlines Date
Eylea Regeneron  sBLA for diabetic retinopathy based on 24-week results.  May 13
Jakafi Novartis/Incyte sNDA for acute GVHD with inadequate response to corticosteroids. May 24
Bydureon Astrazeneca Decision on inclusion of data from Exscel cardiovascular outcome study. May
Botox Allergan sBLA for paediatric upper limb spasticity. Q2 2019
PF-05280586 Pfizer Rituxan biosimilar; FDA accepted filing in September 2018. Q2 2019
Zirabev Pfizer Avastin biosimilar; FDA accepted filing in August 2018.  Q2 2019
Vraylar Allergan sNDA for depressive episodes associated with bipolar disorder. H1 2019
Source: EvaluatePharma. 

In terms of supplementary approvals, Regeneron will hope to add non-proliferative diabetic retinopathy to Eylea's label; Leerink analysts reckon the opportunity could be worth as much as $700m by 2023. Anti-VEGFs have yet to really be used in this setting, which is typically treated with laser surgery, so the hope is that Eylea will meet an unmet need. Company executives have cautioned that it could take time for sales to build in this indication, however. 

Finally, Pfizer and Merck KGaA’s hopes might have been lifted this week by the impressively swift review of Merck & Co’s Keytruda plus Inlyta in first-line renal cell carcinoma. The combination was approved almost two months ahead of a June 20 PDUFA date, just over two months after the application was accepted. 

Bavencio was filed in the same setting, also in combination with Inlyta, with an action date targeted for June, but Keytruda stole its thunder when it generated game-changing data in the Keynote-426 study. Until this readout kidney cancer was thought to be an important market for Bavencio, a checkpoint inhibitor latecomer.

As such, there are reasons to believe that the FDA will be less impressed by Pfizer/Merck KGaA’s Javelin Renal 101 data, meaning that the partners’ might have to wait a little longer to get the agency’s go-ahead (Asco-GU 2019 – checkpoint blockers might not be created equal after all, February 20, 2019).

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