Navigation Links
Tafamidis: Approval denotes proven added benefit
Date:9/17/2012

Tafamidis meglumine (trade name: Vyndaqel) was approved in November 2011 for the treatment of transthyretin amyloidosis in adults. This rare disorder ("orphan disease") is caused by a defective gene and is associated with progressive nerve damage (neurological degeneration) that tafamidis is supposed to delay. According to 35a SGB (Social Code Book) V, an added benefit is regarded as proven if a drug for a rare disease - known as an orphan drug - has been approved. The German Institute for Quality and Efficiency in Health Care (IQWiG) has assessed tafamidis at the request of the Federal Joint Committee (G-BA).

The scientific assessment by IQWiG has produced a hint of a positive effect of tafamidis on neurological degeneration.

Tafamidis compared with "best supportive care"

Transthyretin (TTR) is formed in the liver and is involved in the transport of the thyroid hormone, thyroxine. Mutations in the TTR gene can lead to transthyretin amyloidosis in which there is a build-up of an abnormally modified protein in various organs. These insoluble deposits result in a chronic disease that is associated with the progressive degeneration of the nervous system (polyneuropathy). In most patients, the transthyretin gene is modified at a particular site and this mutation is called "Val30Met". Tafamidis meglumine is approved for the treatment of transthyretin amyloidosis in adult patients with stage 1 neurological disorders to delay the progression of degeneration of peripheral nerves, e.g. in the arms and legs.

Treatment with tafamidis combined with "best supportive care" was compared with "best supportive care" alone. The term "best supportive care" means the therapy that provides the patient with the best possible individually optimized supportive treatment to alleviate symptoms and improve the quality of life, for instance the treatment of pain in polyneuropathy with other drugs (e.g. amitriptyline, gabapentin).

Positive effect in neurological disorders only limited

Only one of the two relevant studies (Fx-005) provided reliable data - and only for patients with a particular genetic defect (Val30Met). The second study (Fx1A-201) in adults with different genetic defects was not controlled, i.e. here the effect of tafamidis was not directly compared with another treatment. It remains unclear how the results of the reliable study can be applied to the patients with genetic defects other than Val30Met.

Assessment of the study data according to the methods of IQWiG produced a hint of a positive effect of tafamidis in respect of the progression of nerve damage. However the effect was only minor.

With regard to other patient-relevant outcomes (other symptoms and side effects, mortality, quality of life), the Institute was unable to identify any further statistically significant advantages or disadvantages of tafamidis compared with the comparator treatment.

G-BA decides on the extent of added benefit

The dossier assessment is part of the overall procedure for early benefit assessment conducted by the G-BA. After publication of the manufacturer's dossier and its assessment by IQWiG, the G-BA initiates a formal commenting procedure which provides further information and can result in a change to the benefit assessment. The G-BA then decides on the extent of the added benefit, thus completing the early benefit assessment.


'/>"/>

Contact: Dr. Anna-Sabine Ernst
presse@iqwig.de
49-221-356-850
Institute for Quality and Efficiency in Health Care
Source:Eurekalert

Related medicine news :

1. Tafamidis: Approval denotes proven added benefit
2. U.S. Bests Canada, Europe in Drug Approvals
3. Approval of Safer Alternative to Pradaxa Delayed by FDA
4. EP Global Communications, Inc. Achieves Shareholder Approval for Restructuring and Signs Definitive Restructuring Agreement
5. UW study plays pivotal role in todays FDA approval of HIV prevention drug
6. Cleveland Clinic Florida receives approval for transplant programs at Weston Facilities
7. Electronic Cigarettes Proven as One of the Most Effective Ways to Quit Smoking
8. Collagenase for Dupuytrens contracture: Added benefit not proven
9. New roadmap suggests proven routes to ending health disparities
10. Added benefit of linagliptin is not proven
11. Benefit of PET and PET/CT in ovarian cancer is not proven
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:2/8/2016)... ... 08, 2016 , ... Stephanie Hebert Insurance Agency, serving families ... new charity campaign. As part of their ongoing community involvement program, funds are ... that children deserve a voice, and in the spirit of neighbors helping neighbors ...
(Date:2/8/2016)... ... 2016 , ... Tingley Rubber Corporation , a leading ... range of unique and advantaged protective solutions to a growing and dynamic ... customer service and marketing support. A new distribution center in Brampton, Ontario will ...
(Date:2/8/2016)... ... February 08, 2016 , ... ... networking and relationship-marketing firm, announced today that nominations will be accepted February ... (ISE®) West Awards. , Awards include the Information Security Executive® of ...
(Date:2/8/2016)... ... 2016 , ... GrassrootsHealth published data from its D*action public ... in the GrassrootsHealth cohort with substantially higher vitamin D levels than a cohort ... states Carole Baggerly, Director of GrassrootsHealth, “the safety and benefits of vitamin ...
(Date:2/8/2016)... (PRWEB) , ... February 08, 2016 , ... The ... annually in this country. The AutismOne 2016 Conference, which is being held May 25-29 ... often won’t hear elsewhere about helpful interventions and causes of chronic illness in children. ...
Breaking Medicine News(10 mins):
(Date:2/8/2016)... 2016 http://www.researchandmarkets.com/research/fqx6nz/global_skin ) ... Skin Protective Equipment Market 2016-2020" report to ... ) has announced the addition of the ... to their offering. --> ... the addition of the "Global Skin Protective ...
(Date:2/8/2016)... , Feb. 8, 2016  A research team ... scientists has discovered details of how the abnormal breakage ... a particularly aggressive form of acute lymphoblastic leukemia (ALL). ... which genetic mutations trigger overproduction of immature cells, called ... The discoveries of the malfunction underlying the type called ...
(Date:2/8/2016)... -- As part of a major growth and expansion initiative, Dune ... Lori Chmura as President of Dune Inc., its U.S. ... extensive experience in the medical device space will play a ... --> --> In her ... marketing and operational functions in the U.S. She is tasked ...
Breaking Medicine Technology: