- Food and Drug Administration introduced breakthrough therapy designation
- Hemophilia drug receives breakthrough therapy designation
- Three cancer drugs also receive the designation
This year, the breakthrough therapy designation (BTD) drugs market is expected to reach $48.8 billion globally, according to BCC Research. By 2022, the market is forecasted to be valued at $99.2 billion, growing at a compound annual growth rate (CAGR) of 15.2%.
The breakthrough therapy designation was introduced by the U.S. Food and Drug Administration in July 2012 as part of the Drug Administration Safety and Innovation Act.
A drug is classified as a BTD if it meets two requirements: It must be a developmental drug devised to treat a serious or life-threatening disease or condition, and preliminary clinical evidence must indicate that the drug demonstrates substantial improvement over existing therapies based on one or more clinical criteria.
Drugs with the BTD designation can reach the market as much as three years sooner than a drug without this designation.
In March 2016, the European Medicines Agency introduced a similar designation called PRIME.
The majority of the therapies approved as BTDs have been oncology drugs, and the main beneficiaries have been pharmaceutical and biotech companies.
Breakthrough Hemophilia Drug
In January, gene therapy company UniQure NV received BTD status for its hemophilia B drug AMT-060.
Hemophilia is a disease in which the body cannot form blood clots. Bleeding can occur following any type of trauma and can be life threatening.
After Phase 1/2 of a clinical trial, the drug was shown to almost completely stop spontaneous bleeding. Prior to tests, the patients had persistent bouts of bleeding which were uncontrolled with preventative infusions.
The patients did not show any serious adverse side effects.
Results were presented in December at the American Society of Hematology’s annual meeting.
The company said it was committed to working with the FDA to bring AMT-060 to the market.
Drug testing is expected to be completed in December 2020.
Other BTDs Show Promise
In late 2016, the FDA approved Venetoclax as a BTD. The drug is used to treat patients with chronic lymphocytic leukemia with 17p deletion. Patients with 17p deletion have a median survival rate and their turmors are resistant to chemotherapy.
Nivolumab and daratumumab also received BTD status.
Nivolumab is for patients with Hodgkin lymphoma. It is the first immune checkpoint inhibitor available for Hodgkin lymphoma. Checkpoint inhibitors have proved successful in treating other types of cancer.
Daratumumab treats multiple myeloma.
- The largest segment of the breakthrough therapy designation is cancer therapy. This year, this segment is expected to reach $19.6 billion. In 2022, it is forecasted to reach $58.6 billion, growing at a CAGR of 24.5%.
- In 2017, the CNS and neurology therapy segment is expected to reach $956 million. By 2022, it is forecasted to reach $8.4 billion, growing at a CAGR of 54.3%.
- Approved breakthrough therapies generated $33.6 billion in 2015. By 2022, new BTDs are forecasted to generate $99.2 billion.