POMPE DISEASE

New treatment for a rare genetic disease

Normally, glycogen-the stored form of glucose-breaks down to release glucose into the bloodstream to be used as fuel for the cells. Patients with Pompe disease have an enzyme deficiency that causes glycogen to accumulate in skeletal and heart muscles, which causes muscle weakness and premature death from respiratory or heart failure.

Recently, the U.S. FDA approved Nexviazyme (avalglucosidase alfa-ngpt) to treat patients 1 year of age and older with late-onset Pompe disease. Nexviazyme, a hydrolytic lysosomal glycogen-specific enzyme, is an I.V. medication that helps reduce glycogen accumulation. The effectiveness of Nexviazyme for the treatment of Pompe disease was demonstrated in a study of 100 patients who were randomized to take Nexviazyme or another FDA-approved enzyme replacement therapy for Pompe disease. Treatment with Nexviazyme improved lung function similar to the improvement seen with the other therapy.

The FDA granted this application Fast Track, Priority Review, and Breakthrough Therapy designations. Nexviazyme also received an orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The most common adverse reactions included headache, fatigue, diarrhea, nausea, arthralgia, dizziness, myalgia, pruritus, vomiting, dyspnea, erythema, paresthesia, and urticaria. Serious reactions included hypersensitivity reactions, such as anaphylaxis, and infusion-associated reactions, such as respiratory distress, chills, and pyrexia. Patients susceptible to fluid volume overload or with compromised cardiac or respiratory function may be at risk for acute cardiopulmonary failure.

"Today's approval brings patients with Pompe disease another enzyme replacement therapy option for this rare disease," said Janet Maynard, M.D., deputy director of the Office of Rare Diseases, Pediatrics, Urologic, and Reproductive Medicine in the FDA's Center for Drug Evaluation and Research.

Sources: http://www.fda.gov/news-events/press-announcements/fda-approves-new-treatment-pohttps://products.sanofi.us/nexviazyme/nexviazyme.pdf

DIABETES

Biosimilar insulin product

Recently approved by the FDA, Semglee (insulin glargine-yfgn) is the first interchangeable biosimilar product approved in the US for the treatment of diabetes. Semglee is a long-acting human insulin analogue indicated to improve glycemic control in adults and pediatric patients with type 1 diabetes mellitus and in adults with type 2 diabetes mellitus. Semglee is both biosimilar to, and interchangeable with, its reference product Lantus (insulin glargine), a long-acting insulin analogue. Approval of insulin products like Semglee can provide patients with additional safe, high-quality, and potentially cost-effective options for treating diabetes.

Semglee is administered subcutaneously once daily and, like Lantus, dosing should be individualized based on the patient's needs and should not be used during episodes of hypoglycemia or in patients with hypersensitivity to insulin glargine products. Also, like Lantus, Semglee is not recommended for treating diabetic ketoacidosis. Semglee may cause serious adverse reactions, including hypoglycemia, severe allergic reactions, hypokalemia, and heart failure. The most common adverse reactions associated with insulin glargine products other than hypoglycemia include edema, lipodystrophy, weight gain, and allergic reactions, such as injection-site reactions, rash, erythema, pain and severe pruritus.

All biological products are approved only after they meet the FDA's rigorous approval standards. The approval of Semglee as biosimilar to, and interchangeable with Lantus, is based on evidence that showed the products are highly similar and that there are no clinically meaningful differences between Semglee and Lantus in terms of safety, purity, and potency. It also showed that Semglee can be expected to produce the same clinical result as Lantus in any given patient and that the risks in terms of safety or diminished efficacy of switching between Semglee and Lantus is not greater than the risk of using Lantus without such switching.

Source: http://www.fda.gov/news-events/press-announcements/fda-approves-first-interchanghttps://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?type=display&setid=9702

IDIOPATHIC HYPERSOMNIA

First-of-its-kind treatment for chronic sleep disorder

Adults with idiopathic hypersomnia (IH) experience chronic excessive daytime sleepiness and often have difficulty waking up from nocturnal sleep or daytime naps. The U.S. FDA recently approved a new indication for Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution for IH in adults. Xywav is a central nervous system depressant previously indicated for the treatment of cataplexy or excessive daytime sleepiness in patients 7 years of age and older with narcolepsy.

The effectiveness of Xywav was evaluated in a double-blind placebo-controlled randomized-withdrawal study in 154 adult patients (ages 19 to 75 years) with IH. In the clinical study, patients who were randomized to switch from Xywav to placebo experienced worsening on measures of sleepiness and symptoms of IH compared with patients randomized to continue treatment with Xywav.

The most common adverse events observed in the study included nausea, headache, dizziness, anxiety, and vomiting. Xywav has a boxed warning for central nervous system depression and abuse and misuse. The active moiety of Xywav is oxybate, also known as gamma-hydroxybutyrate (GHB), a Schedule I controlled substance. Abuse or misuse of illicit GHB has been associated with serious adverse reactions including seizures, respiratory distress, changes in mentation, coma, and death.

Because of the potential risks associated with Xywav, it is subject to strict safety controls on prescribing and dispensing under a program called a Risk Evaluation and Mitigation Strategy. Xywav can be prescribed only by a certified prescriber, and dispensed only to an enrolled patient by a certified pharmacy. Only a certified pharmacy that ships directly to patients can dispense Xywav. Xywav will not be available in retail pharmacies.

The FDA has granted Xywav Orphan Drug, Fast Track, and Priority Review designations.

Sources: https://pp.jazzpharma.com/pi/xywav.en.USPI.pdfhttp://www.uptodate.com/contents/idiopathic-hypersomnia?search=idiopathic%20hype