Tafamidis: Approval denotes proven added benefit

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.

add to favorites email to friend print save as pdf

Related Stories

Belimumab for lupus erythematosus: Added benefit not proven

Aug 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 ...

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

Jan 06, 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 ...

Recommended for you

FDA approves new, hard-to-abuse hydrocodone pill

4 hours ago

Federal health regulators have approved the first hard-to-abuse version of the painkiller hydrocodone, offering an alternative to a similar medication that has been widely criticized for lacking such safeguards.

Soaring generic drug prices draw Senate scrutiny

8 hours ago

Some low-cost generic drugs that have helped restrain health care costs for decades are seeing unexpected price spikes of up to 8,000 percent, prompting a backlash from patients, pharmacists and now Washington ...

Only half of patients take their medications as prescribed

18 hours ago

The cost of patients not taking their medications as prescribed can be substantial in terms of their health. Although a large amount of research evidence has tried to address this problem, there are no well-established ...

Interpol call for roadmap to tackle fake drugs

Nov 19, 2014

Interpol on Wednesday called for a greater global response to pharmaceutical crime as it warned criminal gangs were capitalising on weaknesses in legislation and border controls.

Empagliflozin in type 2 diabetes: Added benefit not proven

Nov 19, 2014

Empagliflozin (trade name Jardiance) has been approved since May 2014 for adults with type 2 diabetes mellitus in whom diet and exercise alone do not provide adequate glycaemic control. The German Institute for Quality and ...

User 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.