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FDA Greenlights Eton and Diurnal’s Orphan Drug for Pediatric Patients
By Judy Ya-Hsuan Lin
Children are vulnerable to many external factors, such as diseases or flu, and internal factors, such as gland secretory deficiency, causing hardships to parents and families. Pharmaceutical companies that develop therapeutics have the challenge of formulating medications differently for adults and children since the latter cannot tolerate high doses.
User-Friendly AI Medication
Approximately 5,000-11,000 pediatric patients in the U.S. suffer from adrenocortical insufficiency (AI). Eton Pharmaceuticals, which specializes in the treatment of rare pediatric diseases, has successfully developed a low dose hydrocortisone formulation, Alkindi Sprinkle, along with UK-based pharmaceutical, Diurnal. AI is a condition in which the adrenal glands do not produce adequate amounts of cortisol and is often caused by Addison’s Disease or Congenital Adrenal Hyperplasia. AI may induce delayed, or stunted physical development, reproductive irregularities and can be potentially fatal. It is a significant cause of morbidity and mortality in children, with 1 in 200 episodes of adrenal crisis, resulting in death.
As recommended by the International Journal of Pediatrics, children with AI should be treated with a 10-12.5 mg dose of hydrocortisone, divided into two to three separate intakes. Previously, the available standard of AI formulation consisted of 5mg of hydrocortisone tablets or even stronger hydrocortisone levels. Pfizer’s Solu-Cortef is another treatment for AI that is administered with intravenous fluid. Thus, the extracted amount can be easily controlled each time, but it is not as user-friendly as oral granules.
The immediate-release oral granule, Alkindi Sprinkle, is an improvement over existing FDA-approved treatments because it saves parents from the nightmare of splitting adult-use hydrocortisone (higher dose) tablets while worrying about overdosing or under-dosing their children.
“We [have] heard from parents about their struggle to provide the right dose to their child [for years],” says Dina M. Matos, Executive Director of the CARES Foundation. Now, Alkindi Sprinkle offers greater flexibility to individualize dosage, as it is available in 0.5 mg, 1 mg, 2 mg, and 5 mg for clinicians. As reported by Diurnal, Alkindi Sprinkle has very high rates of adoption among newly diagnosed AI patients in Europe. With that, the market opportunity is estimated to achieve $100 million.
FDA Approval and Patent Extension Deadline
Based upon six clinical studies, especially the interventional Phase III study of oral hydrocortisone for pediatric AI in newborns to children under eight years old, the FDA has now approved Alkindi Sprinkle, the first oral granule as replacement therapy for AI patients under the age of 17, after the approval throughout Europe in 2018.
Eton expects the drug to be available in the fourth quarter of 2020. In addition to the approval, the FDA granted Alkindi Sprinkle Orphan Drug Designation that accompanies seven years of Orphan Drug Exclusivity. Meanwhile, Diurnal issued three formulations and methods of use patents in the U.S. and extended until 2034. All three patents are expected to be Orange Book listed after the approval.
Sean Brynjelsen, CEO of Eton Pharmaceuticals, said, “Alkindi Sprinkle represents a transformational acquisition for Eton and a major step forward on our journey to become a leader in pediatric rare disease products. This product represents the largest market opportunity within our pipeline and adds a major near-term product launch.”
“The FDA approval of ALKINDI SPRINKLE is a breakthrough for patients and caregivers treating pediatric adrenocortical insufficiency. We are excited to offer an FDA-approved product that enables low dosing and administration of hydrocortisone to pediatric patients. We look forward to making the product available to patients in the coming months.” he added.
Financial Transactions and Collaborations
Eton paid Diurnal US$ 3.5 million and issued 370,474 shares of its common stock (approximately US$ 1.5 million based on an average 15-day trailing stock price). With Orphan Drug Exclusivity granted, Eton will pay a milestone payment of US$ 2.5 million, and Diurnal is entitled to commercial milestone payments based on Alkindi Sprinkle’s achievement of net sale thresholds.
Apart from the financial agreements with Diurnal, Eton’s credit facility with SWK Holdings allows Eton an immediate option to draw US$ 2 million of debt financing and the option to draw an additional US$ 3 million after the FDA approval of Alkindi Sprinkle. The cash will directly support current and future licensing payments to Diurnal and drug-related launch expenses.
Editor: Rajaneesh K. Gopinath, Ph.D.
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