Marinus Pharmaceuticals has received the green light from the Food and Drug Administration for Ztalmy (ganaxolone) oral suspension for the treatment of seizures associated with cyclin-dependent kinase-like 5 deficiency disorder, a rare form of genetic epilepsy, in patients aged 2 years old and older.
“Today is a historic milestone not only for Marinus but for CDD patients, families and caregivers who have long been navigating the unpredictable, often devastating reality of living with uncontrolled seizures,” Scott Braunstein, CEO of Marinus, said. “The approval of Ztalmy would not have been possible without the patients, caregivers and investigators who participated in the clinical trials to develop this important new therapy. We are grateful and humbled by the opportunity to bring the first and only FDA approved treatment for seizures associated with CDD to this community.”
The medication is the first FDA-approved treatment specifically for CDD, is a neuroactive steroid that acts as a positive allosteric modulator of the GABAA receptor, and is expected to be available through a designated specialty pharmacy in July 2022, the company said.
“There has been a great unmet medical need for treatments that address seizures associated with CDKL5 deficiency disorder given their prominent role and profound impact on patients,” principal investigator for the Marigold trial and neurologist and clinical director of Precision Medicine at Children’s Hospital Colorado, Scott Demarest, said. “To date, antiseizure treatment decisions have been based on very limited clinical evidence in this patient population and the resulting outcomes underscore the need for therapies that further improve seizure control. Thanks to our research and this trial, we now have the first treatment specifically approved for seizures associated with CDKL5 deficiency disorder that was shown to have a positive benefit-risk profile.”
“As the mother of a daughter living with CDD, I’ve experienced first-hand the devastating impact seizures can have on these patients,” Karen Utley, president and co-founder of the International Foundation for CDKL5 Research, said. “This approval is monumental for the CDD community—bringing not only the first approved treatment option specifically for CDD patients, but renewed hope to those who have struggled to find medications that are effective in significantly reducing the number of seizures these patients experience on a daily basis.”
Mayne Pharma is welcoming Ann Custin to its board, effective March 23, 2022.
Custin, a United States resident, has almost 40 years of experience in the healthcare sector. Most recently, she was a board director and CFO of Siemens Medical Solutions, now Siemens Healthineers, a leading medical technology company with EUR20b in revenues.
“We are delighted to welcome Ann to the Board of Mayne Pharma,” Mayne Pharma’s chair Frank Condella, said. “She is an experienced executive with a wealth of healthcare commercial experience across the U.S., Europe and Asia. She has extensive experience in strategy, financial planning and reporting, portfolio management, sales operations, manufacturing, risk management, business and corporate development.”
Previously, Custin was chief operating and financial officer of Scient’x Group and president and CEO of USA Draeger Medical Systems.
“I look forward to applying my skills and experience in guiding Mayne Pharma strategically and realizing the potential of its asset base established in both product and pharma service end markets,” Custin said. “I am particularly excited about supporting the board to unlock the value of Mayne Pharma’s businesses for the benefit of shareholders and ensure the company delivers sustainable profitable growth into the future.”
Custin is currently a director of two women’s health-focused companies, Volpara Health and Establishment Labs. She has a Bachelor of Accounting from Queens College and was a Certified Public Accountant.
In addition, she also will join Mayne Pharma’s Audit and Risk Committee, the company said.
Earplanes, known best for their use in helping consumers prevent ear pain while traveling, is adding a brand new feature.
Cirrus Healthcare, the parent company of the brand, announced the launch of the EarPlanes+ companion app, which looks to actively monitor cabin pressure with the smartphone’s barometer.
In addition, the app also sends a notification when pressure is rapidly changing on a flight and gives consumers the knowledge to know when they may need ear protection, the company said.
Available for free, the EarPlanes+ app also gives Cirrus the ability to engage with its consumers directly to help generate in-store sales for its retailers, which include CVS Pharmacy, Target and Walgreens.
The Battle Creek, Mich.-based company announced the launch of its newest flavor, which is inspired by bakery classics.
Snickerdoodle Pop-Tarts feature a snickerdoodle-flavored filling with crunchy cinnamon sugar topping to create a melt-in-your-mouth moment, the company said.
“We know our fans love a ‘dessert for breakfast’ moment, so Pop-Tarts continues to deliver crazy-good flavors that capitalize on this trend with our pie, donut and cake-themed lineups already on shelves,” David Greci, senior brand manager at Pop-Tarts said. “This spring, we’ll complete the cookie trifecta with this delicious new snickerdoodle flavor. Besides, who needs a cookie jar when you've got a toaster and a box of Pop-Tarts?”
Joining the previously launched chocolate chip and cookies and crème varieties, the snickerdoodle Pop-Tarts will be available for purchase in May in an eight-count box that retails for $3.19 at retailers nationwide.
Padagis is introducing a generic version of Tobradex ophthalmic suspension.
The medication is indicated for steroid-responsive inflammatory ocular conditions for which a corticosteroid is indicated and where superficial bacterial ocular infection or a risk of bacterial ocular infection exists.
Tobradex ophthalmic suspension had a market value of roughly $106 million for the 12 months ending January 2022, according to IQVIA.
"This FDA approval and launch demonstrates our commitment to bringing complex, specialized products to patients and customers nationwide who are in search of high quality, affordable specialized healthcare products. The Padagis team partnered with Rafarm to leverage their unique sterile manufacturing skillset in combination with our regulatory and commercial expertise with the overall goal to deliver this important product to the market," Padagis President and CEO Sharon Kochan said.
Oakrum, in collaboration with ANI, recently introduced a generic version of Recordati Rare Diseases’ Cystadane (betaine anhydrous for oral solution) powder in a 180-gram bottle.
The companies were granted Competitive Generic Therapy of 180 days of exclusivity.
The medication is indicated in children and adults for the treatment of homocystinuria to decrease high homocysteine blood levels. Homocystinuria is a rare genetic disorder in which there is an abnormal accumulation of the amino acid homocysteine in the blood and urine. The following are considered to be homocystinuria disorders:
Cystathionine beta-synthase deficiency;
5,10-methylenetetrahydrofolate reductase deficiency; and
Cobalamin cofactor metabolism defect.
“This is the third generic orphan drug that we have launched since starting this business, and we remain committed to bringing rare disease patients affordable options of pharmaceutical products that currently have few or no generic options. Oakrum currently has licensing rights to seven additional ANDAs filed with the FDA and expects to file additional generic orphan drugs later this year,” said Marco Polizzi, CEO of Oakrum Pharma.
“Our collaboration with Oakrum furthers ANI’s mission of bringing high-quality prescription pharmaceutical products to patients in need. Rare diseases are often overlooked, and we are especially pleased to continue identifying patient populations that are underserved and medicines that can help them. This approval with Competitive Generic Therapy status and resulting 180 days of exclusivity marks another milestone for ANI’s R&D engine as a leader in bringing limited competition products to market,” said Nikhil Lalwani, CEO of ANI.