Cranbury Pharma Gets FDA Nod for Generic Emflaza® for Duchenne MD

Cranbury Pharmaceuticals, a subsidiary of Tris Pharma, has received approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA) for a generic version of Emflaza® (deflazacort) oral suspension. This medication is intended for the treatment of Duchenne muscular dystrophy (DMD), a rare genetic disorder characterized by progressive muscle degeneration, primarily affecting boys. The disease typically begins to manifest in early childhood and occurs in about 1 in 5,000 live male births, with an estimated 20,000 children diagnosed globally each year.

Deflazacort oral suspension is now commercially available in the United States for patients aged five and older. Cranbury Pharmaceuticals aims to make this critical therapy more accessible to those with DMD, thus addressing a significant unmet medical need. Janet Penner, the president of Cranbury Pharmaceuticals, expressed pride in launching this essential treatment. She emphasized that Cranbury's mission aligns with Tris Pharma's commitment to developing high-quality medications and ensuring their availability to those in need.

Cranbury Pharmaceuticals was established to market over 20 generic medicines and to advance a diverse pipeline of products capable of treating various disorders and diseases. The company operates from Tris Pharma's headquarters in Monmouth Junction, New Jersey. The FDA approval of the generic deflazacort is a significant milestone, underscoring the critical need for affordable and accessible treatments for DMD. Ketan Mehta, the founder and CEO of Tris Pharma, highlighted the importance of this approval for patients, caregivers, and physicians who rely on such medications.

The launch of Cranbury Pharmaceuticals follows Tris Pharma's recent introduction of Tris Digital Health, a business unit focusing on the development and commercialization of digital diagnostic and therapeutic products for neurological health conditions. These initiatives reflect Tris Pharma's strategy to diversify and expand its portfolio in both digital health and generic medications. Tris Pharma continues to build its commercial portfolio, particularly in the ADHD segment, while advancing its late-stage pipeline of innovative therapeutics for pain, ADHD, neurological disorders, and addiction.

Deflazacort oral suspension has been specifically indicated for the treatment of DMD in patients aged five years and older. However, it comes with significant safety considerations. Patients with known hypersensitivity to deflazacort or its inactive ingredients should avoid this medication. Chronic use of corticosteroids like deflazacort can lead to serious endocrine issues such as Cushing’s syndrome, hyperglycemia, and adrenal insufficiency. Therefore, patients need careful monitoring, especially during times of medical stress or when discontinuing the medication.

Corticosteroids also pose an increased risk of infections, which can be severe and even fatal. Patients should be advised to report any recent or ongoing infections to their healthcare providers. Furthermore, deflazacort can cause cardiovascular and renal issues, including elevated blood pressure and electrolyte imbalances. It should be used cautiously in patients with heart or kidney problems.

Other significant risks include gastrointestinal perforation, psychiatric disturbances, bone density reduction leading to osteoporosis, and ocular issues like cataracts and glaucoma. Long-term use can also negatively impact growth and development in children and increase the risk of thromboembolic events.

The most common adverse reactions to deflazacort include Cushingoid appearance, weight gain, increased appetite, respiratory infections, cough, frequent urination, excessive hair growth, central obesity, and nasopharyngitis. Patients and healthcare providers should be vigilant about these potential side effects and manage them appropriately.

Cranbury Pharmaceuticals, driven by patient-focused innovation, aims to transform new ideas into approved, effective medicines. It leverages Tris Pharma's strengths in product development and commercialization to make high-quality, patient-friendly products widely available.