DUBLIN–(BUSINESS WIRE)–The “Market
Spotlight: Sickle Cell Anemia” report has been added to ResearchAndMarkets.com’s
offering.
This Market Spotlight report covers the sickle cell anemia market,
comprising key marketed and pipeline drugs, clinical trials, recent
events and analyst opinion, upcoming and regulatory events, patent
information, probability of success, a 10-year disease prevalence
forecast, and licensing and acquisition deals.
Key Takeaways
-
The author estimates that in 2017, there were 3.2 million prevalent
cases of sickle cell anemia worldwide, and forecasts that number to
increase to 3.5 million prevalent cases by 2026. -
Africa is estimated to have had 2.7 million prevalent cases of sickle
cell anemia in 2017, accounting for 85% of worldwide cases. The
approved drugs in the sickle cell anemia space predominantly target
fetal hemoglobin, with the exception of Endari, which targets
nicotinamide adenine dinucleotide. These drugs are administered via
the oral route. -
The largest proportion of industry-sponsored drugs in active clinical
development for sickle cell anemia are in Phase II. Therapies in
development for sickle cell anemia focus on a wide variety of targets.
Pipeline drugs are administered via the intravenous or oral routes,
with two products also being tested in subcutaneous formulations. -
High-impact upcoming events in the sickle cell anemia space comprise
an expected CHMP opinion for Endari, a pre-NDA meeting with the FDA
for Voxelotor, topline Phase III trial results for Rivipansel, and
topline Phase II trial results for Olinciguat. -
The overall likelihood of approval of a Phase I hematology asset is
29.4%, and the average probability a drug advances from Phase III is
75.9%. Drugs, on average, take 8.9 years from Phase I to approval in
the overall hematology space. -
There have been 11 licensing and asset acquisition deals involving
sickle cell anemia drugs during 2014-19. The $2,520m strategic
research collaboration deal signed in 2015 between Vertex
Pharmaceuticals and CRISPR Therapeutics for the discovery and
development of drugs based on CRISPR-Cas9 technology was the largest
deal. -
The distribution of clinical trials across Phase I-IV indicates that
the majority of trials for sickle cell anemia have been in the early
and mid-phases of development, with 79% of trials in Phase I-II, and
only 21% in Phase III-IV. -
The US has a substantial lead in the number of sickle cell anemia
clinical trials globally. The UK leads the major EU markets, while
Lebanon has the top spot in Asia. -
Clinical trial activity in the sickle cell anemia space is dominated
by completed trials. Global Blood Therapeutics and Pfizer have the
highest number of completed clinical trials for sickle cell anemia,
with eight trials each. -
Global Blood Therapeutics leads industry sponsors with the highest
number of clinical trials for sickle cell anemia, followed by Novartis.
Topics Covered
OVERVIEW
KEY TAKEAWAYS
DISEASE BACKGROUND
TREATMENT
Bone marrow transplantation
Hydroxyurea
Blood transfusions
Prophylactic therapy
Pain management
EPIDEMIOLOGY
MARKETED DRUGS
PIPELINE DRUGS
RECENT EVENTS AND ANALYST OPINION
LentiGlobin for Sickle Cell Anemia (December 3, 2018)
RVT-1801 for Sickle Cell Anemia (December 3, 2018)
Voxelotor for Sickle Cell Anemia (December 3, 2018)
BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
Voxelotor for Sickle Cell Anemia (June 27, 2018)
Altemia for Sickle Cell Anemia (April 30, 2018)
LentiGlobin for Sickle Cell Anemia (December 9, 2017)
KEY UPCOMING EVENTS
KEY REGULATORY EVENTS
Global Blood Therapeutics Leaves ASH With Momentum Behind Voxelotor In
Sickle Cell Disease
Modus CEO Says Rare Disease Priority Review Voucher To Be Key Catalyst
PROBABILITY OF SUCCESS
LICENSING AND ASSET ACQUISITION DEALS
How Brazilian Patients Might Access Sickle Cell Disease Drug Faster
GBT Picks Up Inclacumab From Roche’s Bargain Bin
EpiDestiny Inc. granted Novo Nordisk AS exclusive global rights to
develop and sell its sickle cell disease candidate EPI01
(decitabine/tetrahydouridine)
PARENT PATENTS
CLINICAL TRIAL LANDSCAPE
Sponsors by status
Sponsors by phase
Recent events
Bluebird ‘Turns A Corner’ With Lentiglobin Gene Therapy, Tests Remain
BIBLIOGRAPHY
Prescription information
APPENDIX
LIST OF FIGURES
Figure 1: Pipeline drugs for sickle cell anemia in the US
Figure 2: Pipeline drugs for sickle cell anemia, by company
Figure 3: Pipeline drugs for sickle cell anemia, by drug type
Figure 4: Pipeline drugs for sickle cell anemia, by drug classification
Figure 5: Voxelotor for Sickle Cell Anemia (December 3, 2018): Phase III
– HOPE
Figure 6: Voxelotor for Sickle Cell Anemia (June 27, 2018): Phase III –
HOPE
Figure 7: Altemia for Sickle Cell Anemia (April 30, 2018): Phase II –
dose finding (SCOT)
Figure 8: Key upcoming events in sickle cell anemia
Figure 9: Probability of success in the sickle cell anemia pipeline
Figure 10: Licensing and asset acquisition deals in sickle cell anemia,
2014-19
Figure 11: Parent patents in sickle cell anemia
Figure 12: Clinical trials in sickle cell anemia
Figure 13: Top 10 drugs for clinical trials in sickle cell anemia
Figure 14: Top 10 companies for clinical trials in sickle cell anemia
Figure 15: Trial locations in sickle cell anemia
Figure 16: Sickle cell anemia trials status
Figure 17: Sickle cell anemia trial sponsors, by phase
LIST OF TABLES
Table 1: Prevalent cases of sickle cell anemia, 2017-26
Table 2: Marketed drugs for sickle cell anemia
Table 3: Pipeline drugs for sickle cell anemia in the US
Table 4: LentiGlobin for Sickle Cell Anemia (December 3, 2018)
Table 5: RVT-1801 for Sickle Cell Anemia (December 3, 2018)
Table 6: Voxelotor for Sickle Cell Anemia (December 3, 2018)
Table 7: BCL11a shRNA(miR) for Sickle Cell Anemia (November 1, 2018)
Table 8: Voxelotor for Sickle Cell Anemia (June 27, 2018)
Table 9: Altemia for Sickle Cell Anemia (April 30, 2018)
Table 10: LentiGlobin for Sickle Cell Anemia (December 9, 2017)
For more information about this report visit https://www.researchandmarkets.com/r/ee14rf
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Related
Topics: Hematology