Navigation Links
Tafamidis: Approval denotes proven added benefit
Date:4/5/2012

This release is available in German.

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: Anna-Sabine Ernst
presse@iqwig.de
49-022-135-6850
Institute for Quality and Efficiency in Health Care
Source:Eurekalert

Related medicine news :

1. Neuralstems NSI-189 trial in major depressive disorder receives FDA approval to advance to Phase Ib
2. FDA Revokes Approval of Avastin for Breast Cancer
3. St. Michaels echocardiology lab awarded international seal of approval
4. Mayo Clinic receives FDA approval for ovarian and breast cancer vaccines
5. Pioneering stem cell bandage receives approval for clinical trial
6. FDA predictability a top concern for medtech firms; EU preferred for product approval
7. New Hepatitis C Drugs Close to Gaining FDA Approval
8. New Hepatitis C Drug Close to Gaining FDA Approval
9. Sanofi Pasteur announces FDA approval of menactra meningococcal conjugate vaccine indication for infants
10. FDA approval of brain aneurysm device gives Jefferson neurosurgeons another life-saving tool
11. Novel immunotherapy drug receives FDA approval for the treatment of metastatic melanoma
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:10/13/2017)... ... October 13, 2017 , ... While it’s often important ... problem. Fortunately, an inventor from Austin, Texas, has identified a solution. , She developed ... darkness or restricted lighting. As such, it eliminates the need to turn on a ...
(Date:10/13/2017)... ... October 13, 2017 , ... Southern ... and Jennifer Huggins, PharmD ’17, along with clinical associate professor Janice Frueh, ... cardiovascular diseases during the 15th Annual Women’s Health Conference. The SIU School ...
(Date:10/13/2017)... ... October 13, 2017 , ... The Visiting Nurse Association (VNA) of Somerset Hills ... specialty vendors and unique items from across the nation, this holiday-themed event will raise ... offered by the VNA. The boutique will be open Saturday, November 4 (10:00 ...
(Date:10/13/2017)... ... October 13, 2017 , ... Talented host, actor Rob Lowe, ... in a new episode of "Success Files," which is an award-winning educational program ... investigates each subject in-depth with passion and integrity. , Sciatica occurs when the ...
(Date:10/12/2017)... ... October 12, 2017 , ... First ... compliance program management, will showcase a range of technology and learning solutions at ... (NCAL) Convention and Expo to be held October 14–18, 2017 at the Mandalay ...
Breaking Medicine News(10 mins):
(Date:9/25/2017)... R.I. , Sept. 25, 2017  EpiVax, ... assessment, vaccine design, and immune-engineering today announced the ... on the development of personalized therapeutic cancer vaccines. ... has provided exclusive access to enabling technologies to ... Eng., MBA will lead EpiVax Oncology as Chief ...
(Date:9/19/2017)... ZirMed Inc ., a recognized leader in cloud-based revenue cycle ... ranked #1 by its users for the seventh consecutive year ... ZirMed was recognized as the top-ranked end-to-end revenue cycle management ... 200 beds and holds one of the longest #1 ranking ... ...
(Date:9/12/2017)... -- Consumer reviews on the independent review site Consumer Affairs ... for hearing aids, ranking it higher than Miracle Ear ™, ... ... Hearing Aids ... store that provides high performance, state-of-the-art, German-engineered hearing aids directly to ...
Breaking Medicine Technology: