Your source for the latest industry trends and drug information news.
Volume 4 | Issue 7
April 1, 2022
Contributors:
Chief Authors: Bonnie Broczkowski & Colleen Peehler
Contributing Author: Ali Zahid, PharmD Candidate; Yi (Eve) Zheng, PharmD/MBA Candidate
On March 18, 2022, the U.S. Food and Drug Administration (FDA) approved Ztalmy® (ganaxolone) oral suspension to treat seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years and older. CDD is a rare genetic disease caused by dysfunction of the CDKL5 gene, which is responsible for making proteins essential for normal brain function and development. CDD patients experience early onset of seizures and suffer from symptoms including poor muscle tone, severe developmental and cognitive delays, delayed or impaired motor skills, reduced visual processing, behavioral abnormalities, as well as difficulties with sleep and digestion. These seizures typically respond poorly to currently available treatments. While it is not known how it causes seizure reduction, Ztalmy® is thought to work by increasing receptors for a chemical called gamma aminobutyric acid type A (GABAA), which can block nerve impulses in the brain. Ztalmy® was evaluated in a double-blind, randomized, placebo-controlled study in patients 2-19 years of age with confirmed CDKL5 gene mutations and a minimum average of 16 convulsive seizures per 28 days that were inadequately controlled by at least two previous treatment regimens. Participants who received Ztalmy® had a 31% reduction in seizures compared to only a 7% reduction for those receiving placebo. The medication has received approval from the FDA with an orphan drug designation, providing another treatment option for patients that are diagnosed with this rare genetic form of epilepsy.
On March 22, 2022, the FDA announced Pfizer’s voluntary recall of certain lots of the blood pressure medication Accuretic™ (quinapril hcl-hydrochlorothiazide). The recall also included two authorized generics distributed by Greenstone due to the presence of a nitrosamine, N-nitroso-quinapril at levels above the Acceptable Daily Intake (ADI). Nitrosamines are organic compounds commonly found in water and foods like cured and grilled meats, dairy products, and vegetables. They pose no risk to human health at low levels, however exposure above an acceptable level for a long period of time may increase the risk of cancer. Quinapril-hydrochlorothiazide is used for the treatment of high blood pressure. Blood pressure control is important to help reduce the risk of fatal and nonfatal cardiovascular events such as strokes and heart attacks. Patients who are taking the recalled products should consult with their providers and pharmacies to determine if they have received the affected product, and alternative treatment options should be discussed with their healthcare provider. Wholesalers and distributors have been advised to stop use and distribution of all affected lot numbers and to notify anyone who has received the recalled product. Patients experiencing any adverse events related to the recall are advised to contact their physician or health care provider. Adverse reactions or quality problems relating to the recall should be reported to the FDA’s MedWatch Adverse Event Reporting Program. To date, Pfizer has not received reports of any adverse events associated with the recall.
On March 15, 2022, the FDA granted approval to Mylan Pharmaceuticals, Inc. for the first generic of the combination product, Symbicort® (budesonide and formoterol fumarate dihydrate) Inhalation Aerosol. Symbicort® is one of the most commonly prescribed products for the treatment of respiratory diseases. It consists of two different ingredients; budesonide (a corticosteroid to reduce inflammation) and formoterol (a long-acting bronchodilator that relaxes muscles in the airways to improve breathing). Symbicort® is approved for use in the treatment of asthma in patients six years and older, and for the maintenance treatment of patients with chronic obstruction pulmonary disease (COPD). Asthma is a chronic lung disease that can start at any age and causes the airways in the lungs to become swollen and extra sensitive to certain environmental “triggers,” like allergens and pollutants. This inflammation makes it difficult to move air in and out of the lungs and causes symptoms such as shortness of breath, chest tightness, coughing or wheezing. COPD (e.g., chronic bronchitis and emphysema) is a chronic, but preventable and treatable disease that causes the airways in the lungs to become inflamed and thickened, which makes it more difficult for the lungs to deliver oxygen to the rest of the body and to remove carbon dioxide waste. This results in symptoms such as chronic cough, shortness of breath, frequent infections, increased production of mucus and wheezing. The budesonide and formoterol inhaler works to improve breathing by opening the airways and reducing inflammation caused by these conditions. It is typically dosed as two inhalations given twice daily, at morning and night about 12 hours apart. It is available in two different strengths; a high dose version which provides 160/4.5 mcg of each product per actuation, and a low dose version which provides 80/4.5 mcg per actuation. Symbicort® was previously available as a branded product and as an authorized generic (same product as the brand name but without the brand label). This approval of generic Symbicort will give providers another treatment option for their patients and increase access to safe, effective, and affordable medications for the consumer.
Novel Drug Approval: Ztalmy® (ganaxolone)
Oral suspension approved for the treatment of seizures associated with a rare developmental epileptic disorder called CDKL5 deficiency disorder (CDD) in patients 2 years and older. [03/18/2022 – Orphan Drug; Priority Review – MARINUS PHARMA INC]
New Dosage Form: Hyftor™ (sirolimus)
Topical gel approved for the treatment of angiofibroma which is characterized by small, red bumps on the face associated with tuberous sclerosis, a genetic disorder that causes skin lesions in patients 6 years and older. [03/22/2022 – Orphan Drug; Priority Review – NOBELPHARMA CO., LTD]
Vimpat® (lacosamide)
Indication: Partial-Onset Seizures
Dosage Form/Strength: 50 MG, 100 MG, 150 MG, and 200 MG Tablets
Average Wholesale Price (AWP): Generic = $534.08 | Brand: $697.85
