Tafamidis: Approval denotes proven added benefit

September 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 , 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 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 in adult patients with stage 1 neurological disorders to delay the progression of degeneration of , 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. , ).

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 . 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.

Explore further: Abiraterone: Indication of considerable added benefit in certain patients

Related Stories

Abiraterone: Indication of considerable added benefit in certain patients

January 6, 2012
Abiraterone (trade name: Zytiga) has been approved since September 2011 for men with metastatic prostate cancer that is no longer responsive to hormone therapy and progresses further during or after therapy with the cytostatic ...

Belimumab for lupus erythematosus: Added benefit not proven

August 29, 2012
Belimumab (trade name Benlysta) has been approved since July 2011 as an add-on therapy for adult patients with the autoimmune disease systemic lupus erythematosus (SLE). This monoclonal antibody is only considered as treatment ...

Added benefit of Cannabis sativa for spasticity due to multiple sclerosis is not proven

September 17, 2012
An extract from the plant Cannabis sativa (trade name Sativex) was approved in May 2011 for patients suffering from moderate to severe spastic paralysis and muscle spasms due to multiple sclerosis (MS). In an early benefit ...

Cabazitaxel can offer an advantage in certain patients

May 29, 2012
Cabazitaxel (trade name: Jevtana) has been approved since March 2011 in men with metastatic prostate cancer who no longer respond to conventional therapy with hormone blockers and have already been pre-treated with the cytostatic ...

Added benefit of linagliptin is not proven

January 6, 2012
Linagliptin (trade name: Trajenta) has been approved since August 2011 to improve blood glucose control ("glycaemic control") in adults with type 2 diabetes mellitus whose elevated blood glucose levels are inadequately controlled ...

Added benefit of fampridine is not proven

August 29, 2012
Fampridine (trade name Fampyra) has been approved in Germany since July 2011 for adult patients suffering from a higher grade walking disability (grades 4 to 7 on the EDSS disability status scale), as a result of multiple ...

Recommended for you

Study suggests ending opioid epidemic will take years

July 20, 2017
The question of how to stem the nation's opioid epidemic now has a major detailed response. A new study chaired by University of Virginia School of Law Professor Richard Bonnie provides extensive recommendations for curbing ...

Team-based model reduces prescription opioid use among patients with chronic pain by 40 percent

July 17, 2017
A new, team-based, primary care model is decreasing prescription opioid use among patients with chronic pain by 40 percent, according to a new study out of Boston Medical Center's Grayken Center for Addiction Medicine, which ...

Private clinics' peddling of unproven stem cell treatments is unsafe and unethical

July 7, 2017
Stem cell science is an area of medical research that continues to offer great promise. But as this week's paper in Science Translational Medicine highlights, a growing number of clinics around the globe, including in Australia, ...

Popular heartburn drugs linked to higher death risk

July 4, 2017
Popular heartburn drugs called proton pump inhibitors (PPIs) have been linked to a variety of health problems, including serious kidney damage, bone fractures and dementia. Now, a new study from Washington University School ...

Most reproductive-age women using opioids also use another substance

June 30, 2017
The majority of reproductive-age and pregnant women who use opioids for non-medical purposes also use at least one other substance, ranging from nicotine or alcohol to cocaine, according to a University of Pittsburgh Graduate ...

At-risk chronic pain patients taper opioids successfully with psychological tools

June 28, 2017
Psychological support and new coping skills are helping patients at high risk of developing chronic pain and long-term, high-dose opioid use taper their opioids and rebuild their lives with activities that are meaningful ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.