FDA Approves Vykat XR (diazoxide choline) to Treat Hyperphagia in Prader-Willi Syndrome

Soleno Therapeutics, Inc.  announced  the U.S. Food and Drug Administration (FDA)  approval of  Vykat XR (diazoxide choline) extended-release tablets, previously referred to as DCCR, for the treatment of hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome (PWS). Soleno expects Vykat XR to be available in the U.S. beginning in April 2025.

“The approval of Vykat XR is a significant milestone for Soleno and, most importantly, for the PWS community who have had no options to treat the most disruptive aspect of this disease,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno. “We are deeply grateful to the many individuals with PWS, their caregivers and clinical sites who participated in our trials, the advocacy groups, including FPWR and PWSA | USA, the advocates who have tirelessly supported the approval of Vykat XR, the FDA for a collaborative review process, and our employees who have been committed to delivering Vykat XR to those with PWS.”

“The FDA approval of Vykat XR is an incredible achievement for the entire PWS community,” said Jennifer Miller, M.D., Professor of Pediatric Endocrinology at the University of Florida, Gainesville, who specializes in treating children and adults with PWS and is a principal investigator in the Vykat XR clinical development program. “I am excited to have Vykat XR available to help treat hyperphagia, which is the most life-limiting aspect of PWS. Families of people with PWS have been prisoners in their own homes because of the need to provide constant, eyes-on supervision 24/7 with access to food being completely restricted.”

“Today marks a historic day for the PWS community. The FDA’s approval of Vykat XR represents a monumental step forward in addressing the longstanding unmet needs of individuals living with PWS and their families,” said Stacy Ward, Chief Executive Officer of the Prader-Willi Syndrome Association | USA. “Our families experience the constant and disruptive challenges of hyperphagia, and Vykat XR offers hope to so many.”

"This approval is a testament to the power of persistence, science, and advocacy," said Susan Hedstrom, Executive Director of the Foundation for Prader-Willi Research. "For years, families and researchers have worked towards a treatment option that truly addresses the complexities of PWS. Today, we take a major step forward in changing the future for individuals navigating hyperphagia associated with PWS."

The FDA approval of Vykat XR was based on an adequate and well-controlled study and safety data from the comprehensive clinical development program. Efficacy was established during the 16-week randomized withdrawal study period of Study 2-RWP (Study C602-RWP), a Phase 3 multi-center, randomized, double-blind, placebo-controlled trial. Individuals randomized to switch to placebo demonstrated a statistically significant worsening of hyperphagia compared with individuals who remained on Vykat XR. Prior to participating in the randomized withdrawal period, all individuals received double-blind and/or open-label Vykat XR for a mean duration of 3.3 years.

Vykat XR has a well-established safety profile with over four years of data across four double-blind and/or open label studies. The primary safety analyses are based on Study 1 (Study C601) and the most common adverse reactions occurring in greater than or equal to 10% of individuals receiving Vykat XR and at 2% greater than placebo included hypertrichosis, edema, hyperglycemia and rash.

Ref: https://en.wikipedia.org/wiki/Diazoxide#:~:text=Diazoxide%2C%20used%20as%20the%20salt,available%20as%20a%20generic%20medication.

FDA Grants Accelerated Approval for Vanrafia (atrasentan) for Proteinuria Reduction in Primary IgA Nephropathy

Novartis  announced the US Food and Drug Administration (FDA)  accelerated approval for Vanrafia® (atrasentan), a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. This is generally defined as a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g1. Vanrafia is a once-daily, non-steroidal, oral treatment that can be added onto supportive care, including a renin-angiotensin system (RAS) inhibitor with or without a sodium-glucose co-transporter-2 (SGLT2) inhibitor.

Vanrafia was granted accelerated approval based on a prespecified interim analysis of the Phase III ALIGN study measuring the reduction of proteinuria at 36 weeks compared to placebo1. It has not been established whether Vanrafia slows kidney function decline in patients with IgAN. The continued approval of Vanrafia may be contingent upon the verification of clinical benefit from the ongoing Phase III ALIGN study evaluating whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 1361. The eGFR data are expected in 2026 and intended to support traditional FDA approval.

“Today’s approval marks an important milestone for people living with IgA nephropathy, offering a new option that can be seamlessly integrated into their existing treatment plan, with no REMS requirement,” said Richard Lafayette, M.D., F.A.C.P., Professor of Medicine, Nephrology and Director of the Glomerular Disease Center at Stanford University Medical Center, and Vanrafia ALIGN Study Investigator and Steering Committee Member. “Vanrafia is a selective ETA receptor antagonist that effectively reduces proteinuria, a major risk factor in IgAN. Taking early, decisive action is critical to help improve outcomes for these patients who too often progress toward kidney failure.”

IgAN is a progressive, rare kidney disease in which the immune system attacks the kidneys, often causing glomerular inflammation and proteinuria10. With almost 13 out of every million people in the US diagnosed per year, it is one of the most common autoimmune kidney diseases, and each person’s journey is unique11,12. Up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring maintenance dialysis and/or kidney transplantation3-10, and response to treatment can vary12,13. Effective, targeted therapies with different mechanisms of action can help physicians select the most appropriate treatment for patients.

REF: https://en.wikipedia.org/wiki/Atrasentan#:~:text=in%20April%202025.-,Names,under%20the%20brand%20name%20Vanrafia.

FDA Approves Mezofy (aripiprazole) Oral Film for the Treatment of Schizophrenia

In continuation of my update on Aripiprazole

CMG Pharmaceuticals, an affiliate of Cha Biotech, announced on the 16th that it had received product approval for the schizophrenia treatment drug Mezofy (formerly Depibzo) from the U.S. Food and Drug Administration (FDA).

Mezofy is an oral film-type schizophrenia treatment drug (ingredient name: aripiprazole) developed by CMG Pharmaceuticals. Patients with mental illnesses including schizophrenia often refuse or spit out medication, but the film-type drug can be taken without water and easily dissolves in the mouth, solving this problem.

CMG Pharmaceuticals first applied for FDA approval in December 2019. At that time, CMG Pharmaceuticals was the world’s first film-type schizophrenia treatment company to knock on the FDA’s door. However, the supplementary inspection was delayed due to problems with overseas raw material factories and the impact of the COVID-19 pandemic.

Meanwhile, Opipza, aripiprazole film treatment from China's LP Pharma, received FDA approval in July of last year. CMG Pharmaceuticals then reapplied for product approval in October of last year and received marketing approval.

Mezofy is the fourth product from a domestic pharmaceutical company to receive FDA approval for an improved new drug. An improved new drug is not simply a copy of a new drug whose patent has expired, but rather an improvement in the dosage method by changing the efficacy or formulation. Unlike simple generic drugs, it is protected separately by a patent.

The company explained, “The price of improved new drugs is set higher than that of generic drugs, and they can be marketed and prescribed under the product name rather than the ingredient name, which is advantageous in increasing awareness in the market.”

CMG Pharmaceuticals expects Mezofy to be launched in the US in the first half of 2026. According to Data Monitor, a US market research and analysis firm, the US schizophrenia treatment market is the largest in the world, at 12 trillion won.

The company predicted that the market for Mezofy would expand to over KRW 22 trillion when its indications are expanded to include bipolar disorder, major depressive disorder, autism spectrum disorder, and Tourette's disorder.

The company said, “We plan to complete the selection of a local distribution partner in the U.S. by the second half of this year,” and “We will work with our distribution partners to establish a competitive drug pricing strategy.”

CMG Pharmaceuticals has set a goal of achieving annual sales of over 100 billion won within five years of entering the U.S. market. CMG Pharmaceuticals CEO Lee Ju-hyung said, “After proving the excellence of Mezofy in the U.S. market, we will advance into other overseas markets.”

At the time of the initial application for product approval, the product name was Depipzo. The company explained that in collaboration with the global pharmaceutical branding specialist Brand Institute, it decided on Mezofy as a name that is easier to remember and less likely to cause prescription errors.

Ref: https://en.wikipedia.org/wiki/Aripiprazole

FDA Approves Atzumi (dihydroergotamine) Nasal Powder for the Acute Treatment of Migraine

Shin Nippon Biomedical Laboratories, Ltd. (TSE:2395),  announced  the U.S. Food and Drug Administration (FDA) approval of    New Drug Application (NDA) for Atzumi™(dihydroergotamine (DHE)) nasal powder for the acute treatment of migraine with or without aura in adults. Atzumi was previously known as STS101.

 

Migraine is a neurological disorder that is thought to be the result of temporary changes in the chemicals, nerves and blood vessels in the brain, with symptoms that are often incapacitating. According to the American Migraine Foundation, approximately 40 million Americans live with migraine. It is the second leading cause of disability worldwide in terms of time lost to disability and most common cause of disability among young women.

"The approval of Atzumi is a milestone to celebrate, providing a new option for the acute treatment of migraine combining long-proven benefits of DHE with a patient-friendly and easy-to-use delivery system developed based on SNBL's novel intranasal drug delivery platform technology," said Dr. Ryoichi Nagata, President and CEO of Satsuma. "We believe that Atzumi will contribute to improving the quality of life of patients struggling for relief from these highly disabling problems."

"DHE plays a unique clinical role in the acute treatment of migraine, providing patients long lasting effects and the unique ability to provide benefit even when taken late in a migraine attack. The convenience of Atzumi, the only DHE nasal powder, will offer patients ease of use combined with the important known DHE clinical advantages", said Dr. Stewart J. Tepper, M.D., Vice President of the New England Institute for Neurology and Headache in Stamford, Connecticut.

About Atzumi

Atzumi is a proprietary drug device product incorporating both Satsuma's advanced nasal powder formulation of dihydroergotamine (DHE) administered via its unique nasal delivery device. The product is designed to provide patients an easy-to-use and easy-to-carry treatment option.

The FDA approval for Atzumi is based on two clinical studies (Phase 1 PK trial and ASCEND Phase 3 open-label, long-term safety trial), which demonstrated fast absorption, rapid achievement of high DHE plasma concentrations, and sustained DHE plasma levels over time as well as safety and tolerability in subjects with migraine.

About Dihydroergotamine (DHE)

Since its approval in 1946, DHE has long been recommended in published migraine treatment guidelines as a first-line acute treatment option for migraine and has significant advantages versus other anti-migraine treatments for many patients. However, disadvantages of current DHE liquid nasal spray and injectable products, including invasive and burdensome administration and/or sub-optimal clinical performance, have limited the widespread use of DHE.

Ref: https://en.wikipedia.org/wiki/Dihydroergotamine

https://pubchem.ncbi.nlm.nih.gov/compound/Dihydroergotamine#section=2D-Structure