WASHINGTON, May 13, 2020 /PRNewswire/ --Vanda Pharmaceuticals, Inc. Inc. (Vanda) (Nasdaq: VNDA) today announced that following the completion of a Type A Meeting with the U.S. Food and Drug Administration (FDA) on May 8, 2020, it has reached agreement with the FDA to resubmit its application for HETLIOZ (tasimelteon) for the treatment of Smith-Magenis Syndrome (SMS). As previously disclosed, Vanda received a Refusal to File letter from the FDA on March 12, 2020. The Type A Meeting resolved the outstanding issues regarding the filing of the application. Vanda plans to resubmit as soon as possible, seeking approval of the solid capsule formulation of HETLIOZ for the treatment of adults with SMS, and the liquid formulation of HETLIOZ for the treatment of children with SMS*.
"We are very pleased with the outcome of the Type A Meeting," said Dr. Mihael H. Polymeropoulos, President and CEO of Vanda. "This meeting was a great example of collaboration with the agency and we appreciate FDA's thoughtful work in helping us advance the progress of our application and bring this potentially important treatment closer to use by patients with SMS."
In December 2018, Vanda reported results of the largest placebo controlled study ever conducted in patients with SMS, with HETLIOZ patients seeing significant improvements in sleep. SMS is a developmental disorder that is frequently caused by a small deletion of human chromosome 17p.1, 2 In some cases, SMS is caused by a point mutation in the RAI1 gene, which resides in the deleted region. SMS is estimated to affect 1 in 15,000-25,000 individuals.3 Patients with SMS present with a number of physical, mental and behavioral issues. The most common symptom of SMS is a severe sleep disorder, which results in significant disruption in the lives of patients and their families.
*Vanda's resubmission will be in the form of a supplemental New Drug Application for the solid capsule formulation of HETLIOZ for the treatment of adults with SMS (the sNDA) and a New Drug Application for the liquid formulation of HETLIOZ for the treatment of children with SMS (the NDA). Originally, Vanda submitted an sNDA covering both formulations, but the FDA requested that Vanda separate that application into an sNDA and an NDA for the different formulations.
References:
1 Williams, SR, Zies, D, Mullegama, SV, Grotewiel, MS, & Elsea, SH. Smith-Magenis syndrome results in disruption of CLOCK gene transcription and reveals an integral role for RAI1 in the maintenance of circadian rhythmicity. Am J Hum Genet. 2012; 90(15376605), 941949.
2 Gropman, AL, Duncan, WC, & Smith, AC. Neurologic and developmental features of the Smith-Magenis syndrome (del 17p11.2). Pediatr Neurol. 2006; 34(08878994), 337350.
3 Greenberg F, Guzzetta V, Montes de Oca-Luna R et al: Molecular analysis of the Smith Magenis syndrome: a possible contiguous gene syndrome associated with del(17)(p11.2). Am J Hum Genet. 1991; 49:1207 1218.
About Vanda Pharmaceuticals Inc.
Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit http://www.vandapharma.comand follow us on Vanda's Twitter and LinkedIn.
Corporate Contact: AJ Jones II Chief Corporate Affairs and Communications Officer Vanda Pharmaceuticals Inc. 202-734-3400 pr@vandapharma.com
Elizabeth Van Every Head of Corporate Affairs Vanda Pharmaceuticals Inc. 202-734-3400 pr@vandapharma.com
CAUTIONARY NOTE REGARDING FORWARD LOOKING STATEMENTS
Various statements in this release, including, but not limited to statements regarding Vanda's HETLIOZ program for the treatment of SMS and plans to resubmit the sNDA and the NDA related to such program, are "forward-looking statements" under the securities laws. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Vanda's forward-looking statements include, among others, Vanda's ability to complete the preparation of and submit the sNDA and the NDA; the FDA's acceptance and review of such filings; Vanda's ability to obtain FDA approval of HETLIOZ for the treatment of SMS in adults and children; and other factors that are described in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Vanda's annual report on Form 10-K for the fiscal year ended December 31, 2019 and quarterly report on Form 10-Q for the quarter ended March 31, 2020, which are on file with the SEC and available on the SEC's website at http://www.sec.gov. In addition to the risks described above and in Vanda's annual report on Form 10-K and quarterly reports on Form 10-Q, other unknown or unpredictable factors also could affect Vanda's results. There can be no assurance that the actual results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Vanda. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved.
All written and verbal forward-looking statements attributable to Vanda or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein. Vanda cautions investors not to rely too heavily on the forward-looking statements Vanda makes or that are made on its behalf. The information in this release is provided only as of the date of this release, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
SOURCE Vanda Pharmaceuticals Inc.
See the article here:
Vanda Pharmaceuticals Announces Agreement with FDA on Resubmission of the Application for HETLIOZ for the Treatment of Patients with Smith-Magenis...
- Rally for Multiple Sclerosis Treatment Funding - May 7th, 2011 [May 7th, 2011]
- Breakthrough Multiple Sclerosis Treatment? - May 8th, 2011 [May 8th, 2011]
- Multiple Sclerosis Treatment - Mayo Clinic - May 11th, 2011 [May 11th, 2011]
- Tracy Macchia: Controversial MS Treatment - May 14th, 2011 [May 14th, 2011]
- What is Multiple Sclerosis - May 19th, 2011 [May 19th, 2011]
- MS Multiple Sclerosis treatment testimonial from Sanoviv Medical Institute - May 20th, 2011 [May 20th, 2011]
- Patient with progressive ms(paraplegia) Fmr treatment 1/4 - May 22nd, 2011 [May 22nd, 2011]
- New Jersey Multiple Sclerosis Treatment with Chiropractic Care - May 23rd, 2011 [May 23rd, 2011]
- MS Cure - Progression - June 2nd, 2011 [June 2nd, 2011]
- CCSVI UK Multiple Sclerosis Liberation Treatment - June 2nd, 2011 [June 2nd, 2011]
- Stem Cell Therapy MS: Sam Harrell and The Superbowl - June 3rd, 2011 [June 3rd, 2011]
- Holly Huber on Recovery: Stem Cell Therapy for Multiple Sclerosis Community Outreach Dallas, TX 2011 - June 8th, 2011 [June 8th, 2011]
- MS treatment sees early success - June 9th, 2011 [June 9th, 2011]
- CCSVI Possible cure for Multiple sclerosis - June 10th, 2011 [June 10th, 2011]
- MS Cure - Introduction to stem cell bone marrow transplant in Australia - June 11th, 2011 [June 11th, 2011]
- (Multiple Sclerosis Cure) Change Your Thinking? (Multiple Sclerosis Cure) - June 12th, 2011 [June 12th, 2011]
- Essiac cancer treatment herbal tincture, Ms Caisse...from The Secrets of Eden - June 12th, 2011 [June 12th, 2011]
- MS walking treatment breakthrough - June 14th, 2011 [June 14th, 2011]
- multiple sclerosis natural treatment | Kathy Canada Testimonial - June 18th, 2011 [June 18th, 2011]
- CCSVI Surgery India - Liberation Procedure Multiple Sclerosis Treatment in India - June 29th, 2011 [June 29th, 2011]
- XCell-Center MS CCSVI Liberation Angioplasty Treatment Results E.Schuurman 18_01_11 - July 1st, 2011 [July 1st, 2011]
- MS Cure - 7PM Project Carmel Turner MS Stem Cell Treatment - July 9th, 2011 [July 9th, 2011]
- MS Update! Tysabri or Gilenya? - July 10th, 2011 [July 10th, 2011]
- What causes BSE, CJD - July 15th, 2011 [July 15th, 2011]
- Kath Bartlett, MS, LAc Balance Method Acupuncture - July 16th, 2011 [July 16th, 2011]
- MS Cure - 730 report ben leahy carmel turner ms stem cell treatment - July 17th, 2011 [July 17th, 2011]
- CCSVI LIberation Treatment Tokuda - July 18th, 2011 [July 18th, 2011]
- FOX News Interview on the O'Reilly Factor - Treatment of Women in Islam - July 18th, 2011 [July 18th, 2011]
- CCSVI treatment bloopers holy crap - July 18th, 2011 [July 18th, 2011]
- Tarah's CCSVI Venoplasty. Dr McGuckin Explaining procedure findings - July 19th, 2011 [July 19th, 2011]
- Cancer Cure: The One-Minute Cure For Cancer, MS - July 24th, 2011 [July 24th, 2011]
- Not Ready to Cheer CCSVI Trials - July 29th, 2011 [July 29th, 2011]
- New Oral Treatment for MS - July 30th, 2011 [July 30th, 2011]
- 27/07/2011 - MS patients seek controversial treatment - August 1st, 2011 [August 1st, 2011]
- MS Cure - 11 I am still walking - August 5th, 2011 [August 5th, 2011]
- Upcoming Treatments for MS - Pt 1 -MSRA Public Lecture - Dr Suzanne Hodgkinson - August 10th, 2011 [August 10th, 2011]
- Stem Cells for MS Community Outreach: Patient Judi Lecoq - Dallas, TX 2011 - August 18th, 2011 [August 18th, 2011]
- Stem Cell Treatment for Multiple Sclerosis - Community Outreach, San Diego: David Oliver - August 19th, 2011 [August 19th, 2011]
- New Study Tests Possible Treatment for MS - August 25th, 2011 [August 25th, 2011]
- Professor Dimitrios Karussis: Treating MS - August 26th, 2011 [August 26th, 2011]
- walking with MS before CCSVI treatment.wmv - August 27th, 2011 [August 27th, 2011]
- Suzy Cohen Discusses The Liberation Treatment for MS - September 14th, 2011 [September 14th, 2011]
- Multiple Sclerosis MS People - Amazing New Treatment - September 15th, 2011 [September 15th, 2011]
- Multiple Sclerosis Alternative Treatment - September 17th, 2011 [September 17th, 2011]
- CLEAR scoliosis treatment, Dr. Culpepper (MS) - September 22nd, 2011 [September 22nd, 2011]
- Liberation treatment for MS - September 23rd, 2011 [September 23rd, 2011]
- 44b - My Lyme disease and rheumatoid arthritis journey - Miraculous CCSVI treatment for MS sufferers - September 24th, 2011 [September 24th, 2011]
- A Closer Look at Gilenya - The First Oral Treatment for MS - September 28th, 2011 [September 28th, 2011]
- Unequal Treatment Drives Disability Rights Movement - September 28th, 2011 [September 28th, 2011]
- Multiple Sclerosis - The Nebraska Medical Center - September 29th, 2011 [September 29th, 2011]
- Testimonial for CCSVI Treatment Excel Heart Center Mexico -- Tanya Reay - September 30th, 2011 [September 30th, 2011]
- Current thinking on what causes MS - Pt 1 - MSRA Public Lecture -Prof Graeme Stewart - October 2nd, 2011 [October 2nd, 2011]
- Current and emerging therapies in MS - Part 02 (How do these treatments work?) - October 2nd, 2011 [October 2nd, 2011]
- MS Richard Humphries before and after treatment for Multiple Sclerosis with stem cells - October 3rd, 2011 [October 3rd, 2011]
- Matthew - CCSVI and his Liberation Treatment - October 8th, 2011 [October 8th, 2011]
- Multiple Sclerosis - Myelin Repair - Video - October 14th, 2011 [October 14th, 2011]
- MS Auto Immune vs CCSVI Treatment Simple Pictures by Berukoff - Video - October 23rd, 2011 [October 23rd, 2011]
- New Promising Treatment for Those Living with Multiple Sclerosis - Video - October 29th, 2011 [October 29th, 2011]
- Stem cells to treat MS - Pt 2 - MSRA Public Lecture - A/Prof Richard Burt - Video - October 30th, 2011 [October 30th, 2011]
- New hope for treating multiple sclerosis - Video - October 31st, 2011 [October 31st, 2011]
- CCSVI Multiple sclerosis Breakthrough/cure - Video - October 31st, 2011 [October 31st, 2011]
- Inhuman Treatment of Wheelchair bound Multiple Sclerosis Patient in Canadian Care Home - Video - November 9th, 2011 [November 9th, 2011]
- MS treatment movie Cells4health.mp4 - Video - November 10th, 2011 [November 10th, 2011]
- Racing to the Cure for MS: Myelin Repair Foundation Celebrates MS Awareness Week - Video - November 12th, 2011 [November 12th, 2011]
- [Symptoms of Multiple Sclerosis] - Video - November 12th, 2011 [November 12th, 2011]
- Multiple Sclerosis Treated with Adult Stem Cells - Video - November 13th, 2011 [November 13th, 2011]
- Stem cells to treat MS - Pt 1 - MSRA Public Lecture - A/Prof Richard Burt - Video - November 16th, 2011 [November 16th, 2011]
- Multiple Sclerosis A Natural Treatment - Video - November 16th, 2011 [November 16th, 2011]
- My MS and Stem Cell Treatment - Video - November 16th, 2011 [November 16th, 2011]
- CCSVI Liberation News Reports - Video - November 20th, 2011 [November 20th, 2011]
- Stem Cells for MS Community Outreach: Patient Sam Harrell - Dallas, TX 2011 - Video - November 21st, 2011 [November 21st, 2011]
- Arthur S13 E2-1 - The Silent Treatment - Video - November 27th, 2011 [November 27th, 2011]
- Holly Huber on Recovery: Stem Cell Therapy for Multiple Sclerosis Community Outreach San Diego CA - Video - December 3rd, 2011 [December 3rd, 2011]
- CCSVI - Video - December 3rd, 2011 [December 3rd, 2011]
- My Sister's Experience with Liberation Treatment for Multiple Sclerosis PART 2/2 - Video - December 5th, 2011 [December 5th, 2011]
- Multiple Sclerosis -- Treatment LDN story - Video - December 7th, 2011 [December 7th, 2011]
- ABC News - Multiple Sclerosis effectively treated by a new acoustic medical device. - Video - December 9th, 2011 [December 9th, 2011]
- MS Patient After Stem Cell Therapy - Video - December 10th, 2011 [December 10th, 2011]
- Multiple Sclerosis - Video - December 12th, 2011 [December 12th, 2011]
- Journey of Connective Tissue, Autoimmune disease, steroid treatment updates. - Video - December 22nd, 2011 [December 22nd, 2011]