What is the criteria for a drug to receive orphan drug designation?
Criteria for Orphan Drug Designation The product must be intended for use in a rare disease or condition. Adequate documentation or prevalence data must demonstrate that the intended condition is rare.
Who makes Sirturo?
TITUSVILLE, NJ, August 9, 2019 — The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that the U.S. Food and Drug Administration (FDA) has granted approval for SIRTURO® (bedaquiline) tablets as part of combination therapy in pediatric patients – those over the age of 12 and younger than 18 and …
Are orphan drugs profitable?
Once approved and marketed, several companies have shown that profits can be made on orphan drugs and patients can be served, despite small numbers of potentially treatable patients. Gross profit margins of over 80% are reported in the rare disease industry, whereas the pharmaceutical industry average is 16%.
How do you get Sirturo?
SIRTURO® is available by prescription from healthcare professionals associated with qualified centers for the evaluation, diagnoses, and treatment of TB, including pulmonary MDR-TB.
Who began manufacturing Sirturo in 2012?
Bedaquiline has been marketed in the United States (accelerated approved 28 December 2012) by its manufacturer Janssen Therapeutics under the proprietary name Sirturo [20].
What is FDA orphan designation?
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US or meets cost recovery provisions of the act.
When did Sirturo become an orphan medicine in the EU?
Tuberculosis is rare in the EU, and Sirturo was designated an ‘ orphan medicine ’ (a medicine used in rare diseases) on 26 August 2005. Further information on the orphan designation : EU/3/05/314. How is Sirturo used? Sirturo can only be obtained with a prescription.
How old do you have to be to take Sirturo?
Sirturo is indicated for use as part of an appropriate combination regimen for pulmonary multidrug resistant tuberculosis (MDR TB) in adults and adolescent patients (12 years to less than 18 years of age and weighing at least 30 kg) when an effective treatment regimen cannot otherwise be composed for reasons of resistance or tolerability.
What does it mean to have conditional authorisation for Sirturo?
Sirturo has been given ‘conditional authorisation’. This means that there is more evidence to come about the medicine, which the company is required to provide. Every year, the Agency will review any new information that becomes available and this overview will be updated as necessary.
What kind of Medicine is Sirturo used for?
This medicine is authorised for use in the European Union. Sirturo is a tuberculosis medicine that contains the active substance bedaquiline. Tuberculosis is an infection caused by the bacterium Mycobacterium tuberculosis.