Posts in category: News

FDA Approves Kebilidi Gene Therapy for the Treatment of AADC Deficiency

PTC Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) accelerated approval of its gene therapy for the treatment of AADC deficiency, the first-ever gene therapy approved in the United States that is directly administered to the brain.

• The gene therapy, which will be marketed in the United States with the brand name Kebilidi™ (eladocagene exuparvovec-tneq), is indicated for the treatment of children and adults with AADC deficiency, including the full spectrum of disease severity.
• AADC deficiency is a highly morbid and life-shortening rare genetic disorder that results in the inability to synthesize dopamine, a neurotransmitter essential for motor function. Kebilidi is a gene replacement therapy that is directly administered to the putamen of the brain through a stereotactic neurosurgical procedure.

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FDA Approves Aucatzyl for Adults with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia

Autolus Therapeutics plc has announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Aucatzyl (obecabtagene autoleucel) for the treatment of adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-ALL).

• Aucatzyl is a CD19-directed genetically modified autologous T cell immunotherapy.
• Aucatzyl is the first CAR T therapy approved by the FDA with no requirement for a REMS program (Risk Evaluation Mitigation Strategy).
• ALL is an aggressive type of blood cancer that can also involve the lymph nodes, spleen, liver, central nervous system, and other organs.

Source: Autolus Therapeutics plc

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November 2024

Exenatide Injection 300mcg/1.2mL (250mcg/mL) and 600mcg/2.4mL (250mcg/mL)
Approved: November 19, 2024 – Amneal Pharmaceuticals LLC
Treatment for: Type 2 Diabetes
Generic for: Byetta

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on May 30, 2024.
Side effects like drowsiness, impaired driving, and respiratory depression (slowed breathing) can worsen when anxiety drugs are combined with alcohol. Many medicines used for anxiety should be used short-term and, in general, you should avoid or limit alcohol with these drugs. Follow the advice from your healthcare provider.

Many anxiety drugs have central nervous system depressant activity and interact with alcohol, so it is
important to understand your risks. A wide variety of medications from different classes, such as
antidepressants or benzodiazepines, are used to treat anxiety disorders.

Source: FDA; Courtesy of Drugs.com

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July 2024

• Indium In-111 Pentetreotide Injection Kit 3 mCi/mL
  Approved: July 1, 2024 – Sun Pharmaceutical Industries, Inc.
  Used for: Diagnosis and Investigation
  Generic for: Octreoscan
• Bupivacaine Liposome Injectable Suspension 133mg/10mL (13.3mg/mL) and 266mg/20mL
  (13.3mg/mL)
  Approved: July 1, 2024 – Jiangsu Hengrui Pharmaceuticals Co., Ltd.
  Used for: Analgesia
  Generic for: Exparel

June 2024

• Palbociclib Tablets 75mg, 100mg and 125mg
  Approved: June 5, 2024 – Synthon Pharmaceuticals, Inc.
  Treatment for: Breast Cancer
  Generic for: Ibrance Tablets
• Avanafil Tablets 50mg, 100mg and 200mg
  Approved: June 14, 2024 – Hetero Labs Limited
  Treatment for: Erectile Dysfunction
  Generic for: Stendra
• Phentermine Hydrochloride and Topiramate Extended-Release Capsules 3.75mg (base)/23mg,
  7.5mg (base)/46mg, 11.25mg (base)/69mg and 15mg (base)/92mg
  Approved: June 25, 2024 – Actavis Laboratories FL, Inc.
  Treatment for: Obesity
  Generic for: Qsymia

FDA Approves Myhibbin Oral Suspension for Prophylaxis of Organ Rejection

Azurity Pharmaceuticals, has announced that the FDA has approved Myhibbin, the only ready-to-use mycophenolate mofetil oral suspension. Mycophenolate mofetil is an antimetabolite immunosuppressant used to protect a donated organ from being rejected from the body’s immune response.

• In 2023, there were over 46,000 transplants in the U.S. and these patients need to take medication every day to fight against rejection.
• Myhibbin is indicated for the prophylaxis of organ rejection in adult and pediatric recipients 3 months of age and older of allogeneic kidney, heart, or liver transplants, in combination with other immunosuppressants.
• It is expected that Myhibbin will become commercially available in pharmacies nationwide in Q2 2024.

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FDA Approves Daybue (trofinetide) for the Treatment of Rett Syndrome

Acadia Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration (FDA) has approved Daybue (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. Daybue is the first and only drug approved for the treatment of Rett syndrome.

Rett syndrome is a complex, rare, neurodevelopmental disorder typically caused by a genetic mutation on the MECP2 gene. It is characterized by a period of normal development until six to 18 months of age, followed by significant developmental regression with loss of acquired communication skills and purposeful hand use. Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities. Rett syndrome is believed to affect 6,000 to 9,000 patients in the U.S.,with a diagnosed population of approximately 4,500 U.S. patients. Daybue is not recommended for patients with moderate or severe renal impairment.

Daybue™ is expected to be available in the U.S. by the end of April, 2023.

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