Questcor's H.P. Acthar Gel sNDA for infantile spasms receives FDA approval

Oct 15 2010

Questcor Pharmaceuticals, Inc. (Nasdaq: QCOR) today announced the U.S. Food and Drug Administration (FDA) has approved Questcor's supplemental new drug application (sNDA) for H.P. Acthar® Gel (repository corticotropin injection) in the treatment of infantile spasms (IS), an ultra-rare orphan disorder affecting approximately 2,000 American children annually.  IS is a devastating and potentially life-threatening form of epilepsy seen in infancy and early childhood.  

"The approval of Acthar for infantile spasms is an example of Questcor's clear focus on helping patients with serious, difficult-to-treat medical conditions," said Don M. Bailey, president and CEO of Questcor.  "Today's approval is an important milestone for babies afflicted with IS, as well as the doctors, nurses and parents who care for them. The FDA approval of Acthar for IS treatment provides an opportunity to begin raising awareness among healthcare providers and parents about the importance of early diagnosis and appropriate treatment of IS.  It will also help to ensure rapid access to Acthar for infants suffering from this devastating condition."

Acthar is already widely used by pediatric neurologists to treat IS based on guidelines published by the American Academy of Neurology and Child Neurology Society.  Many child neurologists, as well as organizations involved in supporting children with IS and their parents, consider Acthar to be a standard of care in the treatment of IS.

"The Child Neurology Foundation is very pleased that the FDA has approved Acthar as a treatment for infantile spasms," said Lawrence W. Brown, MD, president of the Child Neurology Foundation, associate professor of neurology and pediatrics and director of the pediatric neuropsychiatry program at Children's Hospital of Philadelphia. "This is good news for children with this rare form of childhood epilepsy, as well as for parents and families. The FDA approval will also help standardize care for IS by providing specific guidelines for physicians on prescribing Acthar, the drug that has been the most widely-accepted initial approach for IS for more than 50 years."

In conjunction with approval of the IS indication, and as a result of the FDA's orphan designation for Acthar in the treatment of IS, the FDA has also granted Acthar a seven-year exclusivity period during which the FDA is prohibited from approving any other adrenocorticotropic hormone (ACTH) formulation for IS unless the other formulation is demonstrated to be clinically superior to Acthar.  Also, along with the approval notice the FDA has approved a new Acthar label and has finalized a medication guide for Acthar in the treatment of IS. Questcor will provide this guide with each Acthar prescription for IS and assess the guide's usefulness and usage by caregivers of IS patients.

"The product label for Acthar has been thoroughly updated and modernized in conjunction with the review of the sNDA for IS," noted Dr. David Young, Questcor's Chief Scientific Officer.  "In addition to the new indication for IS, the FDA has included other indications where there is evidence or scientific rationale for Acthar efficacy.  For example, the strategically important indications for the treatment of acute exacerbations of multiple sclerosis and for inducing the remission of proteinuria in nephrotic syndrome have remained on the label.  The new label also includes other indications for which the Company believes Acthar may have therapeutic potential, including systemic lupus erythematosus, polymyositis, optic neuritis and others. Importantly, the FDA did not require any post-approval commitments from Questcor for any of the indications on the revamped label beyond those related to the IS medication guide."

FDA approval of the IS indication comes as Questcor is in the process of completing a significant expansion of its Acthar sales force, which will focus on an increased selling effort in MS and the launch of the new IS indication.  The company plans to begin initial educational and promotional efforts based on the new IS indication at the annual meeting of the Child Neurology Society next week in Providence, Rhode Island.

"We have essentially completed the previously announced expansion of our Acthar sales force," Steve Cartt, Questcor's Executive Vice President and Chief Business Officer.  "FDA approval of the new Acthar label, which includes the MS exacerbation indication, reinforces our competitive position in the MS exacerbation market.  Similarly, the Acthar nephrotic syndrome indication, which remains unchanged, will enable us to continue our commercialization plans to address this serious, difficult-to-treat kidney disorder.  New data related to the use of Acthar in the treatment of nephrotic syndrome are expected to be presented at the American Society of Nephrology Annual Meeting in November 2010."

Acthar was originally approved by the FDA in 1952. Prior to the modernization of the Acthar label in connection with FDA approval of the sNDA the label included over 50 approved indications.  Other than the indications for MS exacerbations and nephrotic syndrome, Questcor did not expect to generate any meaningful net sales in the next several years from any of these legacy indications.  The new Acthar label, which will be available in a few days on the Company's website at www.questcor.com or at www.acthar.com, now includes 19 indications organized under the following disease states:

• Infantile spasms: H.P. Acthar Gel (repository corticotropin injection) is indicated as monotherapy for the treatment of infantile spasms in infants and children under 2 years of age.
• Multiple Sclerosis: H.P. Acthar Gel (repository corticotropin injection) is indicated for the treatment of acute exacerbations of multiple sclerosis in adults. Controlled clinical trials have shown H.P. Acthar Gel to be effective in speeding the resolution of acute exacerbations of multiple sclerosis.  However, there is no evidence that it affects the ultimate outcome or natural history of the disease.
• Edematous State: To induce a diuresis or a remission of proteinuria in the nephrotic syndrome without uremia of the idiopathic type or that due to lupus erythematosus.
• Rheumatic Disorders: As adjunctive therapy for short-term administration (to tide the patient over an acute episode or exacerbation) in: Psoriatic arthritis, Rheumatoid arthritis, including juvenile rheumatoid arthritis (selected cases may require low-dose maintenance therapy), Ankylosing spondylitis.
• Collagen Diseases: During an exacerbation or as maintenance therapy in selected cases of: systemic lupus erythematosus, systemic dermatomyositis (polymyositis).
• Dermatologic Diseases: Severe erythema multiforme, Stevens-Johnson syndrome.
• Allergic States: Serum sickness.
• Ophthalmic Diseases: Severe acute and chronic allergic and inflammatory processes involving the eye and its adnexa such as:  keratitis, iritis, iridocyclitis, diffuse posterior uveitis and choroiditis; optic neuritis; chorioretinitis; anterior segment inflammation.
• Respiratory Diseases: Symptomatic sarcoidosis