TW’s Take: if approved, Oleogel-S10 is likely to become Amryt’s largest drug franchise in very short order. Expect to hear about the acceptance and priority review status in Q2. Possible Priority Review Voucher grant in Q4 as well, which would be worth roughly $100M to AMYT.
DUBLIN, Ireland, and Boston MA, March 31, 2021, Amryt (Nasdaq: AMYT, AIM: AMYT), a global, commercial-stage biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, today announces the completion of the rolling submission of a New Drug Application (“NDA”) to the U.S. Food and Drugs Administration (“FDA”) for Oleogel-S10 for the potential treatment of cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (“EB”). EB is a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment.
The NDA submission includes a request for Priority Review which can expedite the review process to six months following acceptance of the NDA submission. Previously Oleogel-S10 has been granted Orphan, Fast Track and Pediatric Rare Disease designation by the FDA. The rolling submission of the NDA began on June 29, 2020.
Based on FDA timelines, Amryt expects to receive notification if Priority Review has been granted and if the NDA has been accepted for filing in Q2 2021.
The NDA is supported by data from the EASE pivotal phase 3 trial in EB (“EASE”). Amryt announced in October 2020 that the EASE study met its primary endpoint of accelerated healing of the target wound by day 45 in patients treated with Oleogel-S10 vs the control gel.
Dr Joe Wiley, CEO of Amryt Pharma, commented: “Our NDA submission to the FDA marks another important milestone for Amryt as we progress our lead development candidate Oleogel-S10 with the regulatory authorities in both the US and Europe. Today’s news follows the recent validation of our MAA for Oleogel-S10 with the EMA. These developments also represent a potentially important advancement for patients and families living with this rare and distressing disorder. We will continue to work closely with the respective regulatory authorities with the hope of bringing Oleogel-S10 to patients as soon as possible.”
* For the purposes of this announcement, we use the product name Oleogel-S10. Filsuvez® has been selected as the brand name for the product but Amryt does not, as yet, have regulatory approval for Filsuvez® to treat EB.
Amryt is a global commercial-stage biopharmaceutical company focused on acquiring, developing and commercializing innovative treatments to help improve the lives of patients with rare and orphan diseases. Amryt comprises a strong and growing portfolio of commercial and development assets.
Amryt’s commercial business comprises two orphan disease products – metreleptin (Myalept®/ Myalepta®) and lomitapide (Juxtapid®/ Lojuxta®).
Myalept® / Myalepta® (metreleptin) is approved in the US (under the trade name Myalept®) as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (GL) and in the EU (under the trade name Myalepta®) as an adjunct to diet for the treatment of leptin deficiency in patients with congenital or acquired GL in adults and children two years of age and above and familial or acquired partial lipodystrophy (PL) in adults and children 12 years of age and above for whom standard treatments have failed to achieve adequate metabolic control. For additional information, please follow this link.
Juxtapid®/ Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medicinal products for adults with the rare cholesterol disorder, Homozygous Familial Hypercholesterolaemia (“HoFH”) in the US, Canada, Columbia, Argentina and Japan (under the trade name Juxtapid®) and in the EU, Israel and Brazil (under the trade name Lojuxta®). For additional information, please follow this link.
Amryt’s lead development candidate, Filsuvez® (Oleogel-S10) is a potential treatment for the cutaneous manifestations of Junctional and Dystrophic Epidermolysis Bullosa (“EB”), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. Filsuvez® has been selected as the brand name for Oleogel-S10. The product does not currently have regulatory approval to treat EB.
Amryt’s pre-clinical gene therapy platform, AP103, offers a potential treatment for patients with Dystrophic EB, and is also potentially relevant to other genetic disorders.
For more information on Amryt, including products, please visit www.amrytpharma.com.
The person making this notification on behalf of Amryt is Rory Nealon, CFO/COO and Company Secretary.
Shore Capital (Edward Mansfield, Daniel Bush, John More) are NOMAD and Joint Broker to Amryt in the UK. Stifel (Ben Maddison) are Joint Broker to the company in the UK.
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