BOTOX. Description. Section: Prescription Drugs Effective Date: January 1, 2013 Subsection: CNS Original Policy Date: December 7, 2011

Transcription

1 Federal Employee Program 1310 G Street, N.W. Washington, D.C Fax Subject: Botox Page: 1 of 6 Last Review Status/Date: December 6, 2012 BOTOX Description Botox (onabotulinum toxin A) Background Botulinum toxin (abbreviated either as BTX or BoNT) is a protein neurotoxin produced by the bacterium Clostridium botulinum. The botulinum toxins are characterized as 7 separate neurotoxins (labeled as types A, B, C [C1, C2], D, E, F, and G), which are antigenically and serologically distinct but structurally similar. The neuromuscular blockade is achieved through prevention of docking/fusion of neurosecretory with the nerve synapse plasma membrane and release of neurotransmitters. The various botulinum toxins have approved cosmetic and non-aesthetic uses. They possess individual potencies, and care is required to assure proper use and avoid medication errors. Recent changes to the established drug names by the FDA were intended to reinforce these differences and prevent medication errors. (1-2) OnabotulinumtoxinA [BoNT-A (BOTOX )] was initially approved in 1989 by the US Food and Drug Administration (FDA) for the treatment of strabismus, blepharospasm, and hemifacial spasm in patients aged younger than 12 years. Subsequently, additional indications have been approved for non-aesthetic uses. OnabotulinumtoxinA is also used for a variety of non-labeled indications for which there is an evidence base and clinical recommendation. (3-11)

2 Subject: Botox Page: 2 of 6 Regulatory Status FDA-approved indication: Botox is an acetylcholine release inhibitor and a neuromuscular blocking agent indicated for: (12) Treatment of urinary incontinence due to detrusor overactivity associated with a neurologic condition [e.g., spinal cord injury (SCI), multiple sclerosis (MS)] in adults who have an inadequate response to or are intolerant of an anticholinergic medication Prophylaxis of headaches in adult patients with chronic migraine ( 15 days per month with headache lasting 4 hours a day or longer) Treatment of upper limb spasticity in adult patients Treatment of cervical dystonia in adult patients, to reduce the severity of abnormal head position and neck pain Treatment of severe axillary hyperhidrosis that is inadequately managed by topical agents in adult patients Treatment of blepharospasm associated with dystonia in patients 12 years of age Treatment of strabismus in patients 12 years of age Important limitations: (12) Safety and effectiveness of Botox have not been established for the prophylaxis of episodic migraine (14 headache days or fewer per month). Safety and effectiveness of Botox have not been established for the treatment of upper limb spasticity in pediatric patients, and for the treatment of lower limb spasticity in adult and pediatric patients. Safety and effectiveness of Botox for hyperhidrosis in body areas other than axillary have not been established. Botulinum toxins are not interchangeable. Related policies Dysport, Myobloc, Xeomin Policy This policy statement applies to clinical review performed for pre-service (Prior Approval, Precertification, Advanced Benefit Determination, etc.) and/or post-service claims.

3 Subject: Botox Page: 3 of 6 Botox may be considered medically necessary for treatment of the conditions indicated below. Botox may be considered investigational for all other indications. Prior Approval Requirements Age 12 years of age or greater except where otherwise indicated below Diagnoses Patient must have ONE of the following: 1. Achalasia 2. Blepharospasm associated with dystonia 3. Cervical dystonia 4. Chronic anal fissures 5. Dysphagia 6. Facial Nerve (VII) disorders 7. Hemifacial spasms 8. Hereditary spastic paraplegia 9. Hyperhidrosis 10. Neuromyelitis optica 11. Orofacial dyskinesia 12. Other dystonia (writer s cramp, focal task specific dystonia, laryngeal) 13. Overactive, neurogenic bladder dysfunction (18 years of age or older) AND ONE of the following: a. Inadequate response to an anticholinergic b. Intolerant of an anticholinergic 14. Prophylaxis of chronic migraine headaches* (18 years of age or older) 15. Spasmodic torticollis (clonic twisting of the head) 16. Spastic hemiplegia 17. Spasticity (upper and lower limbs) due to multiple causes [i.e. cerebral palsy, stroke, multiple sclerosis and post-traumatic brain and spinal cord injury] (no age restriction) 18. Sphincter of Oddi dysfunction 19. Strabismus associated with dystonia 20. Upper limb spasticity (18 years of age or older) *chronic migraine headache defined as having 15 days per month with headache lasting 4 hours a day or longer

4 Subject: Botox Page: 4 of 6 Prior Approval Renewal Requirements Same as above Policy Guidelines Pre PA Allowance None Prior Approval Limits Duration 1 year Prior Approval Renewal Limits Same as above Rationale Summary Botulinum toxin (abbreviated either as BTX or BoNT) is a protein neurotoxin produced by the bacterium Clostridium botulinum. The botulinum toxins are characterized as 7 separate neurotoxins (labeled as types A, B, C [C1, C2], D, E, F, and G), which are antigenically and serologically distinct but structurally similar. The various botulinum toxins have approved cosmetic and non-aesthetic uses. They possess individual potencies, and care is required to assure proper use and avoid medication errors. Recent changes to the established drug names by the FDA were intended to reinforce these differences and prevent medication errors. (1-2) Prior authorization is required to ensure the safe, clinically appropriate and cost effective use of Botox while maintaining optimal therapeutic outcomes. References 1. Blasi J, Chapman ER, Link E, et al. Botulinum neurotoxin A selectively cleaves the synaptic protein SNAP-25. Nature. Sep ;365(6442): Brin MF. Botulinum toxin: chemistry, pharmacology, toxicity, and immunology. Muscle Nerve Suppl. 1997;6:S Zhu Q, Liu J, Yang C. Clinical study on combined therapy of botulinum toxin injection and small balloon dilation in patients with esophageal achalasia. Dig Surg. Feb 2009;26(6): Wehrmann T, Schmitt TH, Arndt A, Lembcke B, Caspary WF, Seifert H. Endoscopic injection of botulinum toxin in patients with recurrent acute pancreatitis due to pancreatic sphincter of Oddi dysfunction. Aliment Pharmacol Ther. Nov 2000;14(11):

5 Subject: Botox Page: 5 of 6 5. Brisinda G, Cadeddu F, Brandara F, et al. Botulinum toxin for recurrent anal fissure following lateral internal sphincterotomy. Br J Surg. Jun 2008;95(6): Greene P, Kang U, Fahn S, Brin M, Moskowitz C, Flaster E. Double-blind, placebocontrolled trial of botulinum toxin injections for the treatment of spasmodic torticollis. Neurology. Aug 1990;40(8): Koman LA, Mooney JF 3rd, Smith BP, Walker F, Leon JM. Botulinum toxin type A neuromuscular blockade in the treatment of lower extremity spasticity in cerebral palsy: a randomized, double-blind, placebo-controlled trial. BOTOX Study Group. J Pediatr Orthop. Jan-Feb 2000;20(1): Annese V, Bassotti G, Coccia G, et al. A multicentre randomised study of intrasphincteric botulinum toxin in patients with oesophageal achalasia. GISMAD Achalasia Study Group. Gut. May 2000;46(5): Simpson D, Gracies J, Graham H, et al. Assessment: Botulinum neurotoxin for the treatment of spasticity (an evidence-based review). Report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology. Neurology 2008;70; Simpson D, Blitzer A, Brashear A, et al. Assessment: Botulinum neurotoxin for the treatment of movement disorders (an evidence-based review). Report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology. Neurology 2008;70: Delgado MR, Hirtz D, Aisen A, et al. Practice Parameter: Pharmacologic treatment of spasticity in children and adolescents with cerebral palsy (an evidence-based review):report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2010;74: Botox. Prescribing Information. Irvine, CA. Allergan Inc. October 2011 Policy History Date December 2011 August 2009 July 2010 Action New Policy On August 3, 2009, the FDA announced it was changing the generic names for both Botox and Myobloc to avoid medication errors. Botox's new generic name is onabotulinumtoxina, after previously being known as botulinum toxin type A. Myobloc's new generic name is rimabotulinumtoxinb, after previously being called botulinum toxin type B. Updated ICD-9 codes, addition of ICD-10 codes, separation of criteria for Botox and Myobloc, and addition of the recently FDA approved diagnosis of spasticity in flexor muscles of the elbow, wrist and fingers for Botox.

6 Subject: Botox Page: 6 of 6 October 2010 September 2011 December 2012 BOTOX (onabotulinumtoxina) for injection is indicated for the treatment of upper limb spasticity in adult patients, to decrease the severity of increased muscle tone in elbow flexors (biceps), wrist flexors (flexor carpi radialis and flexor carpi ulnaris) and finger flexors (flexor digitorum profundus and flexor digitorum sublimis). The efficacy and safety of BOTOX for the treatment of upper limb spasticity were evaluated in three randomized, multi-center, double-blind, placebo-controlled studies. Safety and effectiveness of BOTOX have not been established for the treatment of upper limb spasticity in pediatric patients, and for the treatment of lower limb spasticity in adult and pediatric patients. Updated criteria to mirror newly approved FDA indication for chronic migraine in adults. Updated criteria to mirror newly approved FDA indication for urinary incontinence in people with neurologic conditions such as spinal cord injury and multiple sclerosis who have overactivity of the bladder. Removal of ICD 9 and 10 codes due to lack of specificity. Additional compendial indications for botulinum toxin type A including spasticity (upper and lower limbs) due to multiple causes (i.e. cerebral palsy, stroke, multiple sclerosis and post-traumatic brain and spinal cord injury) in both adults and children, treatment of achalasia in patients who are considered poor candidates for endoscopic dilation or surgery, chronic anal fissure, sphincter of Oddi dysfunction, dysphagia and hyperhidrosis. Annual Review-no change in policy statement. Reference and editorial updates. Keywords This policy was approved by the FEP Pharmacy and Therapeutics Committee on December 6, 2012 and is effective January 1, Signature on File James A. Ferrendelli, M.D.