FDA Approves Gablofen

FDA Approves Movement Disorder Drug from CNS Therapeutics: Gablofen (Baclofen Injection) for the Treatment of Severe Spasticity

ST. PAUL, Minn.--(BUSINESS WIRE)--Nov 23, 2010 - Today, CNS Therapeutics announced the FDA approval of Gablofen (baclofen injection) for use in the management of severe spasticity, giving healthcare providers a new, easy-to-administer and cost-effective intrathecal baclofen treatment option. Severe spasticity is a movement disorder affecting more than 500,000 patients in the U.S. alone and is often brought on by multiple sclerosis, cerebral palsy, spinal cord injury, brain trauma and stroke.

Richard Penn, M.D., a pioneer in intrathecal drug therapies and CNS Therapeutics co-founder and chief scientific officer, implanted the industry's first programmable intrathecal drug pump more than 25 years ago. "Until now, the evolution of intrathecal therapies has been limited to modest innovations in implantable devices," said Penn. "With Gablofen, CNS Therapeutics is driving the industry ahead by developing new options to improve the management of debilitating neurological conditions."

Gablofen is compatible with Medtronic's SynchroMedII programmable drug pumps and is offered in the same standard concentrations as Lioresal Intrathecal (baclofen injection), a drug manufactured by Novartis and marketed exclusively by Medtronic. Gablofen is easy to administer with ready-to-use vials and pre-filled syringes offering clear advantages over glass ampules including a reduction in both refill preparation time and the risk of medicine contamination.

"When we founded CNS Therapeutics, our goal was to improve on existing intrathecal therapies by addressing the concerns of patients and all caregivers involved in the treatment path," said John Foster, CEO of CNS Therapeutics. "Gablofen marks our first FDA approval and is a significant first step in our company mission to innovate in the areas of spasticity, Parkinson's disease and chronic, intractable pain."

CNS Therapeutics was founded to develop and launch new intrathecal therapies and has attracted funding from Thomas, McNerney & Partners and InterWest Partners. In addition to Gablofen, the company is also developing treatments for the management of severe pain, and is collaborating with the University of Helsinki on novel intrathecal therapies for Parkinson's disease. This research is in part funded by a grant from the Michael J. Fox Foundation.

There are approximately 150,000 patients in the U.S. currently implanted with intrathecal drug pumps. "This market has significant growth potential and is ready for improved pharmaceuticals," said Pratik Shah, Ph.D., partner, Thomas, McNerney & Partners. "We have seen innovations from leading manufacturers of intrathecal drug pumps however CNS Therapeutics is currently the only company solely dedicated to innovation of pharmaceuticals administered in these pumps."

"CNS Therapeutics is well-positioned in an industry that has traditionally been dominated by large device manufacturers," said Nina Kjellson, general partner, InterWest. "With a spirit of innovation combined with the ability to adapt and move quickly, we are optimistic about the potential for Gablofen as well as several products to come."

About Gablofen

Gablofen is available in the same standard concentrations as Lioresal (50 mcg/mL, 500 mcg/mL and 2,000 mcg/mL) and offers:

  • Nurses: a convenient, easy-to-administer therapy that features prefilled vials and syringes rather than glass ampules that must be broken to access the medication. This will reduce refill preparation time and the risk of medicine contamination
  • Pharmacists: more streamlined drug preparation and processing, simplified ordering and greater shelf space
  • Patients: a new cost-effective intrathecal baclofen which may result in lower copays and improved access to therapy

Baclofen, originally developed in the 1920s as a potential antiepileptic drug, was also found to be safe and effective for reducing spasticity.1 In the early 1980s, baclofen was discovered to be more effective when delivered intrathecally, a method of delivering the therapy directly and continuously into the central nervous system (CNS) via an implanted drug pump. The use of site-specific drug delivery holds tremendous promise for the treatment of many neurological disorders and pain. Delivering baclofen directly to the CNS avoids the blood brain barrier, results in increased efficacy at dramatically lower doses and significantly reduces the main side effects of systemic (oral) administration. Baclofen intrathecal injection was first approved in 1992 as an orphan drug and is now considered the standard of care for the treatment of severe spasticity of spinal and cerebral origin.2

The number of baclofen treated patients with severe spasticity is expanding by approximately 10,000 new patients per year, and the need for intrathecal baclofen therapy is life long since it does not cure the cause of spasticity.

About Severe Spasticity

Severe spasticity is a movement disorder affecting more than 500,000 patients in the U.S. alone and is often brought on by multiple sclerosis, cerebral palsy, spinal cord injury, brain trauma and stroke. It causes certain muscles to continuously contract, resulting in stiffness or tightness which may interfere with movement, speech and manner of walking.3 There is no cure for spasticity and physicians manage the condition with baclofen, which is delivered orally or intrathecally. In the U.S., approximately 60,000 patients have been treated with intrathecal baclofen. Due to the complexity of this delivery method, CNS Therapeutics developed Gablofen to specifically address the challenges of treating spasticity faced by patients, caregivers and healthcare providers.

About CNS Therapeutics

Founded in 2007 by recognized experts in intrathecal drug therapies, CNS Therapeutics is a private company based in St. Paul, Minnesota. CNS Therapeutics develops and markets safe, high-quality pharmaceuticals for site-specific delivery to the central nervous system to treat neurological disorders and chronic pain. In addition to this first FDA-approved product, Gablofen (baclofen injection), CNS Therapeutics is currently collaborating with U.S. and international scientific experts to develop and test new treatments for pain and Parkinson's disease. For more information, please visit www.cnstherapeutics.com.

Important Gablofen Safety Information

Abrupt discontinuation of intrathecal baclofen, regardless of the cause, has resulted in sequelae that include high fever, altered mental status, exaggerated rebound spasticity, and muscle rigidity, that in rare cases has advanced to rhabdomyolysis, multiple organ-system failure and death.

Prevention of abrupt discontinuation of intrathecal baclofen requires careful attention to programming and monitoring of the infusion system, refill scheduling and procedures, and pump alarms. Patients and caregivers should be advised of the importance of keeping scheduled refill visits and should be educated on the early symptoms of baclofen withdrawal. Special attention should be given to patients at apparent risk (e.g. spinal cord injuries at T-6 or above, communication difficulties, history of withdrawal symptoms from oral or intrathecal baclofen). Consult the technical manual of the implantable infusion system for additional post implant clinician and patient information.

The most common adverse reactions in patients with spasticity of spinal origin were somnolence, dizziness, nausea, hypotension, headache, convulsions and hypotonia. The most common adverse reactions in patients with spasticity of cerebral origin were agitation, constipation, somnolence, leukocytosis, chills, urinary retention and hypotonia.

For complete prescribing information please visit www.gablofen.com/prescribing-info.php

For more information about Gablofen please visit www.gablofen.com

1 http://www.nlm.nih.gov/medlineplus/druginfo/meds/a682530.html; Accessed on 11.22.2010

2 P. Rawlins et. al (2004) ˜Intrathecal Baclofen Therapy Over 10 Years' Journal of Neuroscience Nurses. 36 (6): 322-32

3 http://www.ninds.nih.gov/disorders/spasticity/spasticity.htm; Accessed on 11.22.2010

Gablofen® is a registered trademark of CNS Therapeutics Inc.

Lioresal® is a registered trademark of Novartis Pharmaceuticals Corp.

SynchroMed® is a registered trademark of Medtronic Inc.

Contact: Lois Paul & Partners
Melissa Zipin, 781-782-5726
Melissa_zipin@lpp.com

Posted: November 2010

Gablofen (baclofen) FDA Approval History

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