Breakthrough Therapy Designation

What is a Breakthrough Therapy?

A new drug may be designated as a breakthrough therapy by the Food and Drug Administration (FDA) if it is intended to treat a serious or life-threatening disease and preliminary clinical evidence suggests it provides a substantial improvement over existing therapies. Once the breakthrough therapy designation is requested by the drug sponsor, the FDA and sponsor work together to determine the most efficient path forward.

 

Breakthrough Designations Announced By Companies (as of October 2013):

• Kalydeco - (Vertex) - 2 cystic fibrosis designations
  • in combination with VX-809 in patients with two copies of the F508del mutation 
  • ​monotherapy for other CFTR mutations
• Ibrutinib - (J&J/Pharmacyclics) - 3 designations
  • mantle cell lymphoma
  • waldenstrom macroglobulinemia (WM)
  • chronic lymphocytic leukemia
• LDK378 - (Novartis) - ALK+ NSCLC resistant to crizotinib
• Palbociclib - (Pfizer) - treatment of post-menopausal patients with ER+, HER2- locally advanced or metastatic breast cancer
• Lambrolizumab - (Merck) - inoperable and metastatic melanoma 
• Daclatasvir*- (BMS) - interferon-free treatmemt of Hepatitis C *combination of daclatasvir with two other direct-acting antivirals, asunaprevir, an NS3 protease inhibitor and BMS-791325, an NS5B non-nucleoside polymerase inhibitor
• Daratumumab - (Janssen) - for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD), or who are double refractory to a PI and IMiD
• SD101 - (Scioderm) - topical cream for the treatment of inherited Epidermolysis Bullosa (EB)
• ABT-450* - (AbbVie) - interferon-free 3-DAA treatment of Hepatitis C tested both with and without ribavirin *combination of ABT-450 with two other drugs, ABT-267, an NSA5 inhibitor, and ABT-333, a non-nucleoside polymerase inhibitor
• Obinutuzumab - (Genetech) - for the treatment of chronic lymphocytic leukemia (CLL)
• Sebelipase Alfa - (Synageva) - for the treatment of early onset lysosomal acid lipase deficiency (LAL deficiency)
• Asfotase Alfa - (Alexion Pharmaceutical) - for the treatment of patients with hypophosphatasia (HPP) whose first signs or symptoms occurred prior to 18 years of age, including perinatal-, infantile-, and juvenile-onset forms of the disease
• Serelaxin - (Novartis) - for the treatment of acute heart failure
• Drisapersen - (GlaxoSmithKline) - for the treatment of Duchenne muscular dystrophy (DMD) in ambulant and non-ambulant boys who carry dystrophin gene mutations amenable to exon 51 skipping
• BYM338 (bimagrumab) - (Novartis) - for the treatment of sporadic inclusion body myositis (sIBM)
• Sofosbuvir/ledipasvir combination - (Gilead) - for the treatment of Hepatitis C
• Firdapse (amifampridine phosphate) - (Catalyst Pharmaceutical) - for the symptomatic treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) 
• Entinostat - (Syndax Pharmaceuticals) - in combination with exemestane for the treatment of metastatic ER-positive breast cancer in postmenopausal women who have progressed on hormonal therapy
• Arzerra (ofatumumab) - (Genmab A/S, GlaxoSmithKline) - in combination with chlorambucil for the treatment of previously untreated chronic lymphocitic leukemia (CLL) in patients for whom fludarabine-based therapy is inappropriate 
• Volasertib - (Boehringer Ingelheim) - for the treatment of previously untreated acute myeloid leukemia (AML) in patients over the age of 65
• Alectinib - (Roche) - for the treatment of non-small cell lung cancer 
• MK-5172/MK-8742 - (Merck) - for the treatment of Hepatitis C
• cPMP - (Alexion) - for the treatment of molybdenum cofactor deficiency (MoCD) type A

*As of September 30, 2013, FDA lists 92 total requests for Breakthrough designation, 27 requests granted, and 41 requests denied. FDA does not disclose information regarding specific drugs or sponsors. Currently, about one granted requests has not been publicly announced by their sponsors.

For more info:Frequently Asked Questions: Breakthrough Therapies

FY 2014 CDER BT Totals as of 10/25/13

source: FOCR

Brunei beckons India's pharma, tourism industries

India's pharmaceutical and tourism industries should take a look at the Sultanate of Brunei Darussalam, where the two area hold immense potential for partnership. The island nation of 400,000 people, located on the north coast of the island of Borneo in Southeast Asia, is home to some of the world's virgin rainforests with mega biodiversity. It hosts thousands of species of flowering plants, palms, ferns, orchids, mosses, lichens and liverworts.

Bioprospectors have discovered a wide variety of chemicals in the flora of these tropical forests. The Borneo rainforests are some of the oldest in the world and are a reservoir of genetic diversity which offers a rich source of medicinal plants, high-yield foods, and other useful forest products.

Scientists from around the world are coming to Brunei to discover beneficial chemicals or compounds that occur naturally and which have huge scientific and medical applications.

China is eyeing Brunei to partner in its pharma industry as Borneo has for centuries supplied Chinese traditional medicine with important raw materials.

The Sultanate under the "Wawasan Vision 2035" is seeking a knowledge-based transformation of society. It has adopted a strategy to diversify its economy, based currently on oil and gas resources, and expand business opportunities through promotion of investment, foreign and domestic, both in downstream industries and in clusters beyond the hydrocarbon sector.

The government has identified the sultanate's rich biodiversity and educated workforce as key factors in promoting research and development (R&D) and attracting overseas investors to partner in projects.

Thus, the forestry department is going ahead with plans for a biotechnology industry to develop pharmaceuticals from the microbes found in the rainforests.

Japan-based National Institute of Technology and Evaluation (NITE) and Bruneian scientists are researching the possibilities of using microbes for pharmaceutical purposes.

Germany is another country looking to partner Brunei in pharmaceuticals culled from the Sultanate's diverse plant life.

After speaking to a cross-section of people during a recent visit to the country, this correspondent got the impression that there is a lot of opportunities in the R&D sector and Brunei can be a "valuable partner" for Indian pharma and biotech industry.

Healthcare industry observers point to how the world has in recent years seen a resurgence in the traditional, complementary and alternative system of medicine because of the rising medical cost.

Like elsewhere in Southeast Asia, India's Ayurveda and Siddha systems of medicare are in use in Brunei because of the millennial historical and cultural contacts between India and the region.

Brunei is bound in the north by the South China Sea and surrounded by Malaysia. The coastal position puts Brunei close to vital sea lanes linking the Indian and Pacific Oceans. Brunei's earliest known history dates back to 6th century, when it was called "Puni (a distortion of the Sanskrit "Baruni"), or "Poli". Then a Hindu-Buddhist kingdom, it had linkages with the Sumatran Srivijaya empire and the Majapahit empire of Java, Indonesia.

In the late 14th century, Brunei converted to an Islamic Sultanate when its ruler, Awang Alak Betatar, married a Muslim Johore princess from Malacca, and embraced Islam to become Brunei's first Sultan.

The total population of Brunei is a little over 400,000. About 67 per cent are ethnically Malay, 16 per cent Chinese, almost 12 percent Indian, European or Filipino, and 6 percent indigenous people like the Iban, an ocean-going subgroup of the Dayak, Dusun and the Melanau.

Although Brunei's official language is standard Malay, English is widely spoken and understood as the nation was a British protectorate till 1984.

Oil and gas exports account for the bulk of government revenues. But as reserves are coming down, Brunei in a bid to diversify the economy is marketing itself as a destination for eco-tourism and a financial centre.

Tourism professionals say pristine rainforests, unspoiled coral reefs, mangrove-covered islands, white sand beaches and accessible nature reserves offer visitors an array of eco-tourism options.

The discovery of oil in 1929 had brought a substantial number of Indinto Brunei in the sector and allied services, and later into other professions such as teaching, medicine, engineering, IT and banking.

India established diplomatic relations with Brunei in 1984 after it got independence from Britain. Brunei and India enjoy a fair degree of commonality in their perceptions of major international issues, say officials.

Brunei is a member of the Association of Southeast Asian Nations (ASEAN) and the Asian Pacific Economic Cooperation (APEC).

Brunei supports India's "Look East" policy which aims at broadening its strategic and economic outlook with Southeast Asian nations.

Indian high commissioner L D Ralte said, during a recent visit by Prime Minister Manmohan Singh, there could be greater bilateral collaboration in certain sectors.

"There are areas that we need to concentrate on in order to improve our relationship further, such as direct connections in transport, mutual recognition of education degrees, diversification of our economic relationship (at the moment this is mainly hinged on our petroleum imports from Brunei) and easier movement of manpower between our countries."

There is also room for more cooperation in information and communications technology, particularly private sector collaborations and joint ventures.

Brunei is strategically located at the geographical heart of Southeast Asia. It thus holds the promise of becoming a hub of air, sea and Internet communication between India and the ASEAN countries.

Due to the small size of population and therefore consumer demand, most of the goods from India come into Brunei through re-exports via Malaysia and Singapore. The signing of the ASEAN-India Trade in Services and Investment Agreement in December is expected to boost bilateral trade and investment.

TOI