Amryt Announces Oleogel-S10 EMA Regulatory Update
Amryt Announces Oleogel-S10 EMA Regulatory Update
Ad-hoc expert group to provide independent EB-specific expertise and advice
DUBLIN, Ireland, and Boston MA, January 28, 2022 Amryt (Nasdaq: AMYT), a commercial-stage global biopharmaceutical company dedicated to acquiring, developing and commercializing novel treatments for rare diseases, today announces that the European Medicines Agency (“EMA”) plans to seek independent external epidermolysis bullosa (“EB”) specific expertise and advice on the evaluation of Oleogel-S10 for the treatment of cutaneous manifestations of junctional and dystrophic EB, a rare and distressing genetic skin condition affecting the young children and adults for which there is currently no approved treatment.
The European Medicines Agency (“EMA”) review process for Oleogel-S10 in EB is ongoing and Amryt has responded to outstanding questions. Given the rarity of the disease with no approved treatment, the EMA proposed that an ad hoc expert group, consisting of both EB clinical experts and EB patients, be consulted to provide specific external and independent advice on EB. The opinion of the Committee for Medicinal Products for Human Use (“CHMP”) is now expected in March 2022.
Dr. Joe Wiley, CEO of Amryt Pharma, said: “WWe welcome input from independent EB physicians and, importantly, the voice of EB patients, through the ad hoc expert group to help the EMA advance its regulatory assessment of Oleogel-S10. If approved, we are confident in the potential of Oleogel-S10 to be an effective therapy for patients suffering from this terrible disease and we are ready to launch what will be the first to market new therapy for EB. .
About Epidermolysis Bullosa
Epidermolysis bullosa (EB) is a rare and devastating group of inherited disorders of the skin, mucous membranes, and inner epithelial linings characterized by extreme skin fragility and the development of blisters. Patients with severe forms of EB suffer from severe chronic blistering, ulceration and scarring of the skin, mutilating scars of the hands and feet, joint contractures, strictures of the esophagus and mucous membranes, risk high to develop aggressive squamous cell carcinomas, infections and risk of premature death. The global market opportunity for EB is estimated by the Company at over $1.0 billion.
Amryt is a commercial-stage global 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 activity includes three orphan disease products: metreleptin (Myalept®/ Myalepta®); oral octreotide (Mycapssa®); and lomitapide (Juxtapid®/Lojuxta®).
Myalept®/Myalepta® (metreleptin) is approved in the United States (under the trade name Myalept®) as an adjunct to diet as replacement therapy to treat complications of leptin deficiency in patients with generalized lipodystrophy ( GL) congenital or acquired 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 older and familial or acquired partial lipodystrophy (PL) in adults and children 12 years and older for whom standard treatments have failed to achieve adequate metabolic control. For more information please follow this link.
Mycapssa® (oral octreotide) is approved in the United States for long-term maintenance therapy in patients with acromegaly who have responded to and tolerated treatment with octreotide or lanreotide. Mycapssa® is the first and only FDA-approved oral somatostatin analog. Mycapssa® has also been submitted to the EMA and is not yet approved in Europe. For more information please follow this link.
Juxtapid®/Lojuxta® (lomitapide) is approved as an adjunct to a low-fat diet and other lipid-lowering medications for adults with the rare cholesterol disorder, homozygous familial hypercholesterolemia (“HoFH”) in the United States , in Canada, Colombia, Argentina and Japan (under the trade name Juxtapid®) and in the EU, Israel, Saudi Arabia and Brazil (under the trade name Lojuxta®). For more information please follow this link.
Amryt’s lead development candidate, Oleogel-S10 (Filsuvez®) is a potential treatment for the cutaneous manifestations of junctional and dystrophic epidermolysis bullosa (“EB”), a rare and distressing genetic skin condition affecting young children and adults for which there is currently no approved treatment. Filsuvez® was chosen as the brand name for Oleogel-S10. The product does not currently have regulatory approval to treat EB and is under review by the FDA and EMA.
Amryt’s preclinical gene therapy candidate, AP103, offers a potential treatment for patients with dystrophic EB, and the polymer-based delivery platform has the potential to be developed for the treatment of other genetic disorders.
Amryt also intends to develop oral drugs that are currently only available as injectable therapies through its Transient Permeability Enhancer (TPE®) technology platform. For more information about Amryt, including products, please visit www.amrytpharma.com.
This announcement may contain forward-looking statements and the words “expect”, “anticipate”, “intend”, “plan”, “estimate”, “aim”, “expect”, “project” and similar expressions (or their negative) identify some of these forward-looking statements. The forward-looking statements contained in this announcement are based on numerous assumptions and about Amryt’s current and future business strategies and the environment in which Amryt expects to operate in the future. Forward-looking statements involve inherent known and unknown risks, uncertainties and contingencies because they relate to events and depend on circumstances that may or may not occur in the future and may cause actual results, performance or achievements are materially different from those expressed or expressed. implied by such forward-looking statements. These statements are not guarantees of future performance or the ability to identify and make investments. Many of these risks and uncertainties relate to factors that are beyond Amryt’s ability to control or accurately estimate, such as future market conditions, developments in the COVID-19 pandemic, fluctuations currencies, the behavior of other market participants, the results of clinical trials, the actions of regulators and other factors such as Amryt’s ability to obtain financing, changes in the political, social and regulatory framework in which Amryt operates in either economic, technological or consumer trends or conditions. Past performance should not be taken as an indication or guarantee of future results, and no representations or warranties, express or implied, are made regarding future performance. No one is under any obligation to update or keep current the information contained in this announcement or to provide the recipient with access to any additional relevant information that may arise in connection with it. These forward-looking statements reflect the Company’s current beliefs and assumptions and are based on information currently available to management.
Joe Wiley, CEO / Rory Nealon, CFO/COO, +353 (1) 518 0200, [email protected]
Tim McCarthy, LifeSci Advisors, LLC, +1 (212) 915 2564, [email protected]