MILAN and SAN BRUNO, Calif., March 12, 2019 — Dompé Farmaceutici S.p.A and Dompé U.S. Inc., (collectively Dompé), announced today that the National Organization for Rare Disorders (NORD) has awarded them the 2019 Industry Innovation Award for Oxervate. The Rare Impact Awards celebrate people, organizations, and innovators making exceptional contributions benefitting rare disease patients and caregivers. Oxervate (cenegermin-bkbj) ophthalmic solution 0.002% was approved by the U.S. Food and Drug Administration (FDA) in August 2018 as the first therapy specifically indicated to treat neurotrophic keratitis (NK), a rare and progressive eye disease that can impact the cornea, leading to scarring and reduced visual acuity.1,3
“At NORD, we remain committed to increasing attention on rare diseases that impact day-to-day living for those who suffer from them,” said Peter L. Saltonstall, President and CEO of NORD. “We are proud to honor Dompé with an Industry Innovation Award and to thank them for the work they are doing on behalf of the rare disease community.”
Dompé supports the rare disease community through continued research and development activity in rare diseases, support of NORD’s RareCare® program, and membership on NORD’s corporate council.
“Dompé is extremely proud of the development program that resulted in the FDA approval of Oxervate. In neurotrophic keratitis, we saw a devastating disease without any approved pharmacologic treatments targeting the root pathogenesis. We are honored to be able to offer this breakthrough therapy to patients who have been waiting for an effective and non-invasive option,” said Sergio Dompé, Chairman of Dompé. “We would like to thank NORD for the Industry Innovation Award. We are inspired by our patients and their caregivers, and we remain committed to innovation for the benefit of patients facing even the rarest of conditions.”
The Rare Impact Awards will take place on Saturday, June 22 at Space Center Houston, the Official Visitor Center of NASA Johnson Space Center and a Smithsonian Affiliate.
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases. NORD is committed to the identification, treatment, and cure of the more than 7,000 rare diseases, of which approximately 90% are still without an FDA-approved treatment or therapy. Rare diseases affect 25-30 million Americans. More than half of those affected are children.
NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. For more than 35 years, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies and education, advancing medical research, and providing patient and family services for those who need them most. NORD represents more than 280 disease-specific member organizations and their communities and collaborates with many other organizations in specific causes of importance to the rare disease patient community.
Oxervate: Safety and Efficacy Data
The efficacy and safety of Oxervate in Neurotrophic Keratitis (NK) was established in two independent, double-masked, randomized, multi-center, controlled clinical trials.2,4 This program represents the largest combined population of patients with NK ever examined in randomized controlled trials. Both trials studied Oxervate (20 mcg/mL) as compared to vehicle among patients with moderate (Stage 2) or severe (Stage 3) NK. Study NGF0212 (REPARO), which was conducted in Europe, randomized 52 patients to each group. REPARO also tested 10mcg/ml as compared to vehicle. After eight weeks, 72.0 percent of patients in the 20mcg/mL group were completely healed vs 33.3 percent in the vehicle group after 8 weeks. Study NGF0214, conducted in the U.S., randomized 24 patients to each group, and 65.2 percent in the 20mcg/mL group were completely healed vs 16.7 percent in the vehicle group after 8 weeks. Both studies included a follow-up period, in REPARO it was 48 weeks, and in Study NGF0214 it was 24 weeks. In REPARO, the study with the longer follow-up, approximately 80 percent of patients who healed during the eight-week treatment period remained healed after one year.1,2
Oxervate was well tolerated in clinical trials, adverse effects were mostly local, mild, and transient.1,2 Eye pain was the most frequent adverse reaction reported in 16% of patients.1 Investigators reported that restoring corneal innervation and sensitivity (which, in turn, will promote corneal healing) can be associated with increased ocular surface symptoms.3 Other adverse events reported in 1-10% of patients included corneal deposits, foreign body sensation, ocular hyperemia, ocular inflammation and tearing.1 Patients should remove contact lenses before applying Oxervate and wait 15 minutes after instillation of the dose before reinsertion.1 Findings from the REPARO trial program are published in the September 2018 issue of Ophthalmology, the journal of the American Academy of Ophthalmology. Topline data from the confirmatory NGF0214 study were presented at the 2017 Congress of the European Society of Ophthalmology, and trial results are in preparation for publication.
About Neurotrophic Keratitis
Neurotrophic keratitis, also known as neurotrophic keratopathy, is a rare disease that can be caused by damage to any level of the fifth cranial (trigeminal) nerve. Causes of impaired corneal sensation include herpetic or other infections, ocular or neurologic surgeries, physical injury to the ocular surface, and some systemic conditions that impair sensation. Diagnosis, prognosis, and treatment are based on disease severity, which is classified into three stages. Stage 1 (mild) exhibits ocular surface irregularity and blurred vision, stage 2 (moderate) exhibits a recurrent persistent epithelial defect (PED), and stage 3 (severe) exhibits corneal ulceration involving subepithelial (stromal) tissue, which may progress to corneal melting and perforation. Neurotrophic keratitis affects fewer than 65,000 iniduals in the U.S. based on epidemiological data on conditions associated with NK.3 Patients with advanced stages of neurotropic keratitis are typically treated by cornea specialists.
The cornea is the most densely innervated tissue in humans, with approximately 7,000 nerve endings per square millimeter. These nerves are unique in providing support to the cornea, which is crucial to help maintain transparency in this tissue that is normally devoid of blood vessels. In fact, corneal nerves help maintain ocular surface health by producing soluble factors called neurotrophins and facilitating sensory-dependent blinking and tearing reflexes.5
About Oxervate (Cenegermin-bkbj) Ophthalmic Solution
Oxervate is a topical solution 0.002% of cenegermin-bkbj (20 mcg/mL) administered every two hours, six times per day, for eight weeks for the treatment of neurotrophic keratitis. Its active ingredient is cenegermin, a recombinant form of human nerve growth factor. Nerve growth factor (NGF) acts through specific high-affinity and low-affinity nerve growth factor receptors in the anterior segment of the eye to support corneal innervation and integrity.
More information about Oxervate can be found in the full prescribing information on the product: www.oxervate.com. Oxervate is now commercially available in the U.S. and can be ordered through Accredo Specialty Pharmacy by calling 1-877-831-8112.
Important Safety Information
The most common adverse reaction in clinical trials that occurred more frequently with Oxervate was eye pain (16% of patients). Adverse reactions included corneal deposits, foreign body sensations in the eye, ocular hyperemia (enlarged blood vessels in the white of the eyes), swelling (inflammation) of the eye, and increase of tears (1-10% of patients).
Contact lenses should be removed before applying Oxervate because the presence of a contact lens (either therapeutic or corrective) could theoretically limit the distribution of cenegermin-bkbj onto the area of the corneal lesion. Lenses may be reinserted 15 minutes after administration.
Oxervate may cause mild to moderate eye discomfort such as eye pain during treatment. The patient should be advised to contact their doctor if a more serious eye reaction occurs.
Dompé is a private, rapidly scaling global biopharmaceutical company founded in Milan, Italy, with a 165-year legacy of medical innovation. Today, Dompé employs 700 employees worldwide and is growing as we enter new markets. In the United States, the commercial operations hub is located in the San Francisco Bay Area, and the company maintains an R&D presence in Boston.
Forward Looking Statements
This press release makes reference to certain information that may not coincide with expected future results. Dompé firmly believes in the soundness and reasonableness of the concepts expressed. However, some of the information is subject to a certain degree of indetermination in relation to its research and development activities and the necessary verifications to be performed by regulatory bodies. Therefore, as of today, Dompé cannot guarantee that the expected results will be consistent with the information provided above.
1 U.S. Prescribing Information.
2 Bonini S, Lambiase A, Rama P, Sinigaglia F, Allegretti M, Chao W, Mantelli F, for the REPARO Study Group. Phase II Randomized, Double-Masked, Vehicle-Controlled Trial of Recombinant Human Nerve Growth Factor for Neurotrophic Keratitis. Ophthalmology. 2018;125:1332-43.
3 Sacchetti M, Lambiase A. Diagnosis and Management of Neurotrophic Keratitis. Clinical Ophthalmology. 2014;8:571-9.
4 Chao W, Benitez Del Castillo JM, Dana R, Geerling G, Mantelli F, Massaro-Giordano G, Rama P. Healing of Persistent Epithelial Defects or Corneal Ulcer by Recombinant Human Nerve Growth Factor Eye Drops in Patients with Stage 2 or 3 Neurotrophic Keratitis. Presented at the Congress of the European Society of Ophthalmology (SOE), Barcelona, Spain, June, 10–13, 2017.
5 Mastropasqua L, Massaro-Giordano G, Nubile M, Sacchetti M. Understanding the Pathogenesis of Neurotrophic Keratitis: The Role of Corneal Nerves. Journal of Cellular Physiology. 2017;232(4):717-24.