Your source for the latest industry trends and drug information news.
Volume 6 | Issue 23
December 1, 2024
Contributors:
Chief Authors: Rebecca Waite, PharmD
Contributing Authors: Salome Mawi, PharmD/MBA Candidate and Xintian Wu, PharmD/MBA Candidate
On November 14, 2024, the U.S. Food and Drug Administration (FDA) approved Kebilidi (eladocagene exuparvovec-tneq), a gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. AADC is a rare genetic neurological disorder that can affect the production of chemical messengers that allow cells in the nervous system to communicate with each other. Affected individuals may experience decreased muscle tone, decreased motor control and movement, and developmental and cognitive delays. Kebilidi is the first FDA-approved gene therapy for the treatment of AADC deficiency. Kebilidi is administered as four infusions during one surgical session into a specific area of the brain. Due to the complexity of the administration, it should be administered in a medical center that specializes in pediatric neurosurgery that have the ability to use imaging and special equipment to deliver the treatment into the brain. After successful administration, Kebilidi treatment results in increased production of dopamine, a critical neurotransmitter in the brain associated with movement, attention, learning and memory.
The approval was based on an open-label, single-arm clinical trial among 13 pediatric patients with the most severe presentation AADC deficiency. The motor assessments were completed for 12 of the 13 patients at week 48 after receiving the treatment. The data showed that gross motor function improved in 8 of 12 treated individuals. These results have not been reported in untreated patients with this disorder. Kebilidi is contraindicated in patients who have not achieved skull maturity assessed by neuroimaging. Common side effects include involuntary muscle movements, fever, low blood pressure, low red blood cell count, increased saliva production, insomnia and respiratory and cardiac arrest.
On November 19, 2024, Endo, Inc. expanded its previously announced voluntary recall of clonazepam orally disintegrating tablets. The product is being recalled due to the mislabeling of cartons with the incorrect strength. Clonazepam is indicated for the treatment of seizures and panic disorders. Use of the mislabeled product could expose patients to a higher or lower dose than intended. Using a higher dose of clonazepam increases the risk of significant sedation, dizziness, impaired coordination, confusion and respiratory depression. Using a lower dose may lead to symptoms that are normally managed with the medication, such as seizures or panic symptoms.
The recalled clonazepam orally disintegrating tablets are packaged in cartons containing 60 blister-packed tablets. Endo is providing written notification to wholesalers and retailers to arrange for the return of the recalled lot. Consumers with the recalled products should stop using the product and contact their health care provider. Adverse effects should also be reported to the FDA’s MedWatch Adverse Event Reporting program. To date, Endo has not received any reports of adverse events related to this recall.
On November 8, 2024, the FDA approved Aucatzyl® (obecabtagene autoleucel) for the treatment of relapsed or refractory adult B-cell acute lymphoblastic leukemia (ALL). Acute lymphoblastic leukemia is a type of blood cell cancer with symptoms that include fatigue, easy bleeding or bruising and enlarged lymph nodes. Aucatzyl® is prepared using the patient’s own blood cells that are collected and genetically modified to target and destroy the cancer cells that contain a specific target, called CD19.
The approval was based on an open-label, multicenter, single-arm trial that enrolled adults with relapsed or refractory CD19-positive B-cell ALL. 27 of the 65 patients achieved complete remission within 3 months after Aucatzyl® infusion. The prescribing information contains a boxed warning for cytokine release syndrome (CRS), T-cell malignancies and a neuropsychiatric syndrome known as immune effector cell-associated neurotoxicity syndrome (ICANS). The most common side effects associated with Aucatzyl® include musculoskeletal pain, viral infections, fever, nausea, bacterial infectious disorders and diarrhea.
New Drug: Imuldosa™ (ustekinumab-srlf)
Subcutaneous (under the skin) or intravenous injection, approved as biosimilar to Stelara® for the treatment of multiple inflammatory autoimmune disorders, including plaque psoriasis, psoriatic arthritis, Crohn’s disease and ulcerative colitis.
Stendra® (avanafil)
Indication: For the treatment of erectile dysfunction.
Dosage Form/Strength: 50mg, 100mg, 200mg tablets
Average Wholesale Price (AWP): Generic = $422 | Brand = $469
