FDA Approves Repository Corticotropin Injection for Infantile Spasms

Megan Brooks

October 19, 2010

October 19, 2010 — The US Food and Drug Administration (FDA) on Friday approved the Questcor Pharmaceuticals Inc supplemental new drug application for the use of repository corticotropin injection (Acthar Gel) as monotherapy for infantile spasms in infants and children younger than 2 years.

The action follows a May 11, 2010, meeting, in which members of the FDA Peripheral and Central Nervous System Drugs Advisory Committee overwhelmingly agreed by a vote of 22 to 1, with 1 abstention, that Questcor had provided substantial evidence of the drug's effectiveness "from a single, adequate, and well-controlled clinical trial with confirmatory evidence from 2 other studies."

Acthar Gel is a natural adrenocorticotropic hormone (ACTH) designed to provide prolonged release after intramuscular or subcutaneous injection. It has been used unofficially for more than 50 years by neurologists to treat a variety of disorders, including multiple sclerosis and seizures.

Current guidelines from the American Academy of Neurology and Child Neurology Society state that ACTH is “probably effective for the short-term treatment of infantile spasms” (Level B evidence). A summary of the guidelines is available at http://aan.com/professionals/practice/pdfs/infantile_spasms_clinicians.pdf.

In an interview with Medscape Medical News, Lawrence W. Brown, MD, president of the Child Neurology Foundation, said, “It's remarkable that it has taken over 50 years to approve a standard drug for this severe, devastating seizure disorder of early infancy, in which time is of the essence. While there are other options, in most studies this drug has proven to be the most frequently effective.”

Dr. Brown, an associate professor of neurology and pediatrics and director of the pediatric neuropsychiatry program at Children's Hospital of Philadelphia in Pennsylvania, said the FDA approval will also help “standardize care for [infantile spasms] by providing specific guidelines for physicians on prescribing Acthar.”

Infantile spasm is a rare form of epilepsy. It is estimated that there are fewer than 2000 new cases of the disorder in the United States each year, giving infantile spasm orphan disease status. Infantile spasm typically begins in infancy between 3 and 6 months of age and is characterized by sudden, brief, massive muscle jerks — typically head drops with associated outstretched arms.

Initially, the attacks are often brief, infrequent, and not typical, leading often to a delay in diagnosis. Because of the pattern of the attacks and the cry that a child gives during or after an attack, the attacks are sometimes initially thought to be due to colic or gastric distress.

Toxicity Concerns

During the May 2010 advisory committee meeting, Phillip L. Pearl, MD, professor of pediatrics and neurology at the George Washington University School of Medicine in Washington, DC, and division chief of General Child Neurology at Children's National Medical Center, voiced concern about the potential toxicity of Acthar Gel.

He and other members of the panel suggested that a registry be established to formally keep track of adverse events and safety with Acthar Gel. Others suggested additions to the package insert.

"It would be helpful to be clear in the package insert about what needs to be monitored. Blood pressure, electrolytes, brain atrophy, cardiomyopathic changes, these things need to be clearly stated,” Dr. Pearl said at the meeting.

According to Questcor, along with the approval of Acthar Gel for infantile spasms, the FDA has also approved a new label for the drug and has finalized a medication guide for Acthar in the treatment of infantile spasms. Questcor says it will provide this guide with each Acthar prescription for infantile spasms and assess the guide’s usefulness and use by caregivers of patients with infantile spasms.

In addition to infantile spasms, Acthar Gel is currently approved in the United States for the treatment of acute exacerbations of multiple sclerosis, nephrotic syndrome, and numerous other diseases and disorders.

Dr. Brown discloses that the Child Neurology Foundation, for which he serves as president, has partnerships that Questcor, which supports infantile spasms awareness week and an infantile spasms research award.

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