2019 Market Spotlight on Sickle Cell Anemia – ResearchAndMarkets.com

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