Overview
Sickle Cell Disease is treated with 6 medications in our database, including OXBRYTA, DROXIA, HYDREA, Hydroxyurea, L-Glutamine, and 1 more. 4 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Pfizer, Bristol Myers Squibb, Emmaus. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Sickle Cell Disease treatment below.
Also known as:
Autosomal recessive
Passed on when both parents carry the same gene change; often skips generations
Infantile
Begins in infancy, roughly 1 month to 2 years old
FDA & Trial Timeline
10 eventsUniversity Hospital, Rouen — PHASE3
St. Jude Children's Research Hospital
Emory University — PHASE4
University Hospital, Tours — NA
Fulcrum Therapeutics — PHASE2
Assistance Publique - Hôpitaux de Paris — PHASE1, PHASE2
Mayo Clinic — NA
CSL Behring — PHASE2
Sanofi — PHASE3
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
8 availableLyfgenia
1 INDICATIONS AND USAGE LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events. LYFGENIA is an autologous hematopo…
1 INDICATIONS AND USAGE LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events. LYFGENIA is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events. ( 1 ) Limitations of Use Following treatment with LYFGENIA, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions. LYFGENIA has not been studied in patients with more than two α-globin gene deletions. ( 1 ) Limitations of Use Following treatment with LYFGENIA, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions [see Adverse Reactions (6.1) ] . LYFGENIA has not been studied in patients with more than two α-globin gene deletions.
CASGEVY
1. INDICATIONS AND USAGE CASGEVY is indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises transfusion-dependent β - thala…
1. INDICATIONS AND USAGE CASGEVY is indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises transfusion-dependent β - thalassemia (TDT) CASGEVY is an autologous genome edited hematopoietic stem cell-based gene therapy indicated for the treatment of patients aged 12 years and older with: sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs). ( 1 ) transfusion-dependent β-thalassemia (TDT). ( 1 )
ADAKVEO
1 INDICATIONS AND USAGE ADAKVEO ® is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease. ADAKVEO is a s…
1 INDICATIONS AND USAGE ADAKVEO ® is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease. ADAKVEO is a selectin blocker indicated to reduce the frequency of vasoocclusive crises in adults and pediatric patients aged 16 years and older with sickle cell disease.
DROXIA
1 INDICATIONS AND USAGE DROXIA is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to sever…
1 INDICATIONS AND USAGE DROXIA is indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises. DROXIA is an antimetabolite indicated to reduce the frequency of painful crises and to reduce the need for blood transfusions in patients with sickle cell anemia with recurrent moderate to severe painful crises. ( 1 )
Endari
1 INDICATIONS AND USAGE Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older. ENDARI is an amino acid indicated to redu…
1 INDICATIONS AND USAGE Endari is indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older. ENDARI is an amino acid indicated to reduce the acute complications of sickle cell disease in adult and pediatric patients 5 years of age and older. ( 1 )
OXBRYTA
1 INDICATIONS AND USAGE OXBRYTA is indicated for the treatment of sickle cell disease (SCD) in adults and pediatric patients 4 years of age and older. This indication is approved under accelerated app…
1 INDICATIONS AND USAGE OXBRYTA is indicated for the treatment of sickle cell disease (SCD) in adults and pediatric patients 4 years of age and older. This indication is approved under accelerated approval based on increase in hemoglobin (Hb) [see Clinical Studies (14) ] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). OXBRYTA is a hemoglobin S polymerization inhibitor indicated for the treatment of sickle cell disease in adults and pediatric patients 4 years of age and older. This indication is approved under accelerated approval based on increase in hemoglobin (Hb). Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). ( 1 )
FERRIPROX
treatment of transfusional iron overload in adult and pediatric patients 8 years of age and older with sickle cell disease
L-Glutamine
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersNational Institutes of Health Clinical Center
📍 Bethesda, Maryland
👤 Payal P Khincha, M.D.
👤 Christopher Grunseich, M.D.
Arkansas Children's Hospital
📍 Little Rock, Arkansas
Boston Children's Hospital
📍 Boston, Massachusetts
Massachusetts General Hospital
📍 Boston, Massachusetts
👤 Matthew Frigault, MD
👤 Janssen Research & Development, LLC Clinical Trial
University of Michigan
📍 Ann Arbor, Michigan
👤 Janssen Research & Development, LLC Clinicaltrial
University of California San Diego
📍 La Jolla, California
Riley Hospital for Children
📍 Indianapolis, Indiana
Children's National Hospital
📍 Washington D.C., District of Columbia
Financial Resources
14 resourcesThe Assistance Fund — Sickle cell disease
The Assistance Fund
Sickle cell disease
Patient Services Inc — Sickle cell disease
Patient Services Inc
Sickle cell disease
Good Days — Sickle cell disease
Good Days
Sickle cell disease
HealthWell Foundation — Sickle cell disease
HealthWell Foundation
Sickle cell disease
NORD Patient Assistance — Sickle cell disease
NORD Patient Assistance
Sickle cell disease
Patient Advocate Foundation — Sickle cell disease
Patient Advocate Foundation
Sickle cell disease
PAN Foundation — Sickle cell disease
PAN Foundation
Sickle cell disease
8 travel grants are also available for Sickle cell disease patients — see Travel Grants below ↓
Travel Grants
8 grantsEmmaus Life Sciences Commercial Co-Payment Assistance Program for Endari
Emmaus Life Sciences
This program provides financial assistance to commercially insured patients aged five and older who are prescribed Endari for sickle cell disease. Applicants must meet undisclosed financial eligibility requirements and submit a Patient Financial Assistance Application.
PAN: Sickle Cell Disease (Medicare)
Patient Access Network Foundation
Patients must have Medicare or Medicaid insurance and a valid sickle cell disease diagnosis to qualify for this $2,000 annual benefit. The program is restricted to residents of the United States and Puerto Rico and is defined as a needs-based assistance fund.
Sickle Cell Disease Financial Assistance Program
The Assistance Fund
The Assistance Fund provides financial assistance to patients and families for out-of-pocket medical costs like copayments, coinsurance, and deductibles. Eligibility is generally based on having a diagnosis covered by their specific disease programs and demonstrating financial need.
Maryland Sickle Cell Disease Association Transportation Assistance Program
Maryland Sickle Cell Disease Association
The Assistance Fund - Sickle Cell Disease (SCD) Financial Assistance
Community
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4 articlesCaregiver Resources
NORD Caregiver Resources
Support, advocacy, and financial assistance for caregivers of rare disease patients.
Mental Health Support
Rare disease caregiving can be isolating. Connect with counseling and peer support.
Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Sickle cell disease
What is Sickle cell disease?
Sickle Cell Disease is treated with 6 medications in our database, including OXBRYTA, DROXIA, HYDREA, Hydroxyurea, L-Glutamine, and 1 more. 4 of these have manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Pfizer, Bristol Myers Squibb, Emmaus. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Sickle Cell Disease treatment below.
How is Sickle cell disease inherited?
Sickle cell disease follows a autosomal recessive inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
At what age does Sickle cell disease typically begin?
Typical onset of Sickle cell disease is infantile. Age of onset can vary across affected individuals.
Are there clinical trials for Sickle cell disease?
Yes — 20 recruiting clinical trials are currently listed for Sickle cell disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Sickle cell disease?
25 specialists and care centers treating Sickle cell disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Sickle cell disease?
6 FDA-approved treatments and 14 patient support programs are currently tracked on UniteRare for Sickle cell disease. See the treatments and support programs sections for copay assistance, eligibility, and contact details.