bluebird bio Confirms Participation in Center for Medicare and Medicaid Innovation (CMMI) Cell and Gene Therapy Access Model
bluebird bio (NASDAQ: BLUE) has reached an agreement with the Center for Medicare and Medicaid Innovation (CMMI) to offer an outcomes-based agreement for LYFGENIA gene therapy under the Cell and Gene Therapy Access Model. The therapy is approved for sickle cell disease patients aged 12 and older with a history of vaso-occlusive events.
Currently, over half of all states have confirmed coverage for LYFGENIA, with more than 50% of Medicaid-insured sickle cell disease patients living in states that have approved at least one prior authorization. The model will enroll patients for 6 years, with individual patient tracking for 5 years. States have until February 28, 2025, to opt into the program, with participation beginning between January 2025 and January 2026.
bluebird bio (NASDAQ: BLUE) ha raggiunto un accordo con il Centro per l'Innovazione Medicare e Medicaid (CMMI) per offrire un accordo basato sui risultati per la terapia genica LYFGENIA nell'ambito del Modello di Accesso alla Terapia Cellulare e Genica. La terapia è approvata per i pazienti affetti da malattia falciforme di età pari o superiore a 12 anni con una storia di eventi vaso-occlusivi.
Attualmente, oltre la metà degli stati ha confermato la copertura per LYFGENIA, con oltre il 50% dei pazienti affetti da malattia falciforme assicurati da Medicaid che vivono in stati che hanno approvato almeno un'autorizzazione preventiva. Il modello registrerà i pazienti per 6 anni, con monitoraggio individuale dei pazienti per 5 anni. Gli stati hanno tempo fino al 28 febbraio 2025 per aderire al programma, con partecipazione che inizierà tra gennaio 2025 e gennaio 2026.
bluebird bio (NASDAQ: BLUE) ha llegado a un acuerdo con el Centro para la Innovación de Medicare y Medicaid (CMMI) para ofrecer un acuerdo basado en resultados para la terapia génica LYFGENIA bajo el Modelo de Acceso a Terapias Celulares y Génicas. La terapia está aprobada para pacientes con enfermedad de células falciformes de 12 años o más con antecedentes de eventos vaso-oclusivos.
Actualmente, más de la mitad de todos los estados han confirmado la cobertura para LYFGENIA, con más del 50% de los pacientes con enfermedad de células falciformes asegurados por Medicaid viviendo en estados que han aprobado al menos una autorización previa. El modelo inscribirá pacientes durante 6 años, con seguimiento individual de pacientes durante 5 años. Los estados tienen hasta el 28 de febrero de 2025 para optar por el programa, con participación que comenzará entre enero de 2025 y enero de 2026.
bluebird bio (NASDAQ: BLUE)는 LYFGENIA 유전자 치료에 대한 결과 기반 계약을 제공하기 위해 Medicare 및 Medicaid 혁신 센터(CMMI)와 협정을 체결했습니다. 이 치료법은 혈색소병 환자 중 12세 이상으로 혈관 폐쇄 사건의 병력이 있는 환자에게 승인되었습니다.
현재, 50% 이상의 주에서 LYFGENIA에 대한 보장을 확인했으며, Medicaid 보험에 가입한 혈색소병 환자의 50% 이상이 최소한 하나의 사전 승인을 승인한 주에 살고 있습니다. 이 모델은 6년 동안 환자를 등록하며, 5년 동안 개별 환자 추적을 진행합니다. 주에서는 2025년 2월 28일까지 프로그램에 참여하기로 선택할 수 있으며, 참여는 2025년 1월과 2026년 1월 사이에 시작됩니다.
bluebird bio (NASDAQ: BLUE) a conclu un accord avec le Centre pour l'Innovation Medicare et Medicaid (CMMI) pour proposer un accord basé sur les résultats pour la thérapie génique LYFGENIA dans le cadre du Modèle d'Accès aux Thérapies Cellulaires et Génétiques. La thérapie est approuvée pour les patients atteints de drépanocytose âgés de 12 ans et plus ayant des antécédents d'événements vaso-occlusifs.
Actuellement, plus de la moitié des états ont confirmé la couverture pour LYFGENIA, avec plus de 50 % des patients drépanocytaires assurés par Medicaid vivant dans des états ayant approuvé au moins une autorisation préalable. Le modèle inscrira des patients pendant 6 ans, avec un suivi individuel des patients pendant 5 ans. Les États ont jusqu'au 28 février 2025 pour se porter candidats au programme, avec une participation débutant entre janvier 2025 et janvier 2026.
bluebird bio (NASDAQ: BLUE) hat eine Vereinbarung mit dem Zentrum für Medicare und Medicaid Innovation (CMMI) getroffen, um eine ergebnisorientierte Vereinbarung für die Gentherapie LYFGENIA im Rahmen des Zugangsmodells für Zell- und Gentherapie anzubieten. Die Therapie ist für Patienten mit Sichelzellanämie ab 12 Jahren geeignet, die eine Vorgeschichte von vaso-okkusive Ereignisse haben.
Derzeit haben über die Hälfte aller Bundesstaaten die Deckung für LYFGENIA bestätigt, wobei mehr als 50% der Medicaid-versicherten Patienten mit Sichelzellanämie in Bundesstaaten leben, die mindestens eine vorherige Genehmigung erteilt haben. Das Modell wird Patienten für 6 Jahre anmelden, wobei die individuelle Patientenverfolgung 5 Jahre dauert. Die Bundesstaaten haben bis zum 28. Februar 2025 Zeit, um sich für das Programm anzumelden, wobei die Teilnahme zwischen Januar 2025 und Januar 2026 beginnen wird.
- Over 50% of states have confirmed LYFGENIA coverage through Preferred Drug Lists
- Majority of Medicaid-insured sickle cell patients live in states with LYFGENIA authorization
- Company secured agreement for outcomes-based payment model with CMMI
- Program participation is voluntary, creating uncertainty about state adoption rates
- Implementation delayed until January 2025
Insights
This agreement with CMMI represents a significant development for bluebird bio's market access strategy for LYFGENIA. The outcomes-based agreement model addresses two critical aspects: financial risk management for state Medicaid programs and patient access to expensive gene therapy.
The current coverage statistics are noteworthy - over 50% of states have already confirmed LYFGENIA coverage and more than half of Medicaid-insured sickle cell patients live in states with at least one approved authorization. The addition of fertility preservation support through the Model eliminates a key access barrier.
The 5-year patient tracking period for outcomes demonstrates a robust approach to value-based healthcare, while the February 2025 opt-in deadline gives states adequate time to evaluate participation. This structured approach to Medicaid access could significantly impact LYFGENIA's commercial success, particularly given the high proportion of sickle cell patients covered by Medicaid.
This policy development could substantially improve LYFGENIA's commercial prospects. With a current market cap of just
- Reduced reimbursement uncertainty for state Medicaid programs
- Streamlined access for a significant portion of the target patient population
- Built-in performance metrics that align payment with clinical outcomes
The 6-year enrollment window provides a substantial timeframe for market penetration, while the outcomes-based structure helps mitigate financial risks for state programs. This could accelerate adoption rates and improve predictability of revenue streams, particularly important for a company of bluebird's size focusing on high-cost gene therapies.
Company and CMMI agreed to key terms to be offered to State Medicaid Agencies to support access to LYFGENIA™ gene therapy for sickle cell disease
States can opt in to an outcomes-based agreement with bluebird through the Model until February 28, 2025
“Ensuring timely, equitable access to gene therapy for people living with sickle cell disease insured through Medicaid has been a cornerstone of our commercial approach since approval, and we are pleased to build on this commitment by offering an outcomes-based agreement to state Medicaid agencies through the Cell and Gene Therapy Access Model,” said Tom Klima, Chief Commercial & Operating Officer at bluebird bio. “We are also pleased to be able to offer fertility preservation support to eligible patients insured by Medicaid in states that opt into the Model, as we recognize these services address an important potential barrier to access.”
Participation in the CGT Access Model builds on bluebird’s established leadership in developing and implementing innovative outcomes-based agreements and has the potential to further strengthen the coverage landscape for LYFGENIA. To date, more than half of all states have affirmed coverage for LYFGENIA through Preferred Drug Lists or published coverage policies, and over
bluebird currently offers two outcomes-based agreements for LYFGENIA to State Medicaid Agencies, and the CGT Access Model may be a bandwidth extender for states that are unable to implement outcomes-based agreements on their own or in a timely fashion. bluebird’s offering through the CGT Access Model is rooted in its validated principles of meaningful risk sharing for payers, and tying performance to clinically meaningful outcomes that are easily tracked through established data sources over a defined period of time.
The model will enroll patients for a period of 6 years and individual patients will be followed for 5 years to track performance-related outcomes. Participation in the CGT Access Model is voluntary and open to all states that participate in the Medicaid Drug Rebate Program (MDRP). With key terms in place, states have until February 28, 2025 to opt-in to participate and states may begin participation in the Model between January 2025 and January 2026.
About LYFGENIA™ (lovotibeglogene autotemcel) or lovo-cel
LYFGENIA is a one-time ex-vivo lentiviral vector gene therapy approved for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events (VOEs). LYFGENIA works by adding a functional β-globin gene to patients’ own hematopoietic (blood) stem and progenitor cells (HSPCs). Durable production of adult hemoglobin with anti-sickling properties (HbAT87Q) is possible following successful engraftment. HbAT87Q has a similar oxygen-binding affinity to wild-type HbA, limits sickling of red blood cells and has the potential to reduce VOEs.
The Phase 1/2 HGB-206 study of LYFGENIA is complete and the Phase 3 HGB-210 study evaluating LYFGENIA is ongoing. bluebird bio is also conducting a long-term safety and efficacy follow-up study (LTF-307) for patients with sickle cell disease who have been treated with LYFGENIA in bluebird bio-sponsored clinical studies.
Indication
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 (VOEs).
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.
Important Safety Information
Boxed WARNING: HEMATOLOGIC MALIGNANCY
Hematologic malignancy has occurred in patients treated with LYFGENIA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 6 months and through integration site analysis at Months 6, 12, and as warranted.
Hematologic Malignancy
Hematologic malignancy has occurred in patients treated with LYFGENIA (Study 1, Group A). At the time of initial product approval, two patients treated with an earlier version of LYFGENIA using a different manufacturing process and transplant procedure (Study 1, Group A) developed acute myeloid leukemia (AML). One patient with α-thalassemia trait (Study 1, Group C) has been diagnosed with myelodysplastic syndrome (MDS).
The additional hematopoietic stress associated with mobilization, conditioning, and infusion of LYFGENIA, including the need to regenerate the hematopoietic system, may increase the risk of a hematologic malignancy. Patients with sickle cell disease have an increased risk of hematologic malignancy as compared to the general population.
Patients treated with LYFGENIA may develop hematologic malignancies and should have lifelong monitoring. Monitor for hematologic malignancies with a complete blood count (with differential) at least every 6 months for at least 15 years after treatment with LYFGENIA, and integration site analysis at Months 6, 12, and as warranted.
In the event that a malignancy occurs, contact bluebird bio at 1-833-999-6378 for reporting and to obtain instructions on collection of samples for testing.
Post-Marketing Long Term Follow-Up Study: Patients who intend to receive treatment with LYFGENIA are encouraged to enroll in the study, as available, to assess the long-term safety of LYFGENIA and the risk of malignancies occurring after treatment with LYFGENIA by calling bluebird bio at 1-833-999-6378. The study includes monitoring (at pre-specified intervals) for clonal expansion.
Delayed Platelet Engraftment
Delayed platelet engraftment has been observed with LYFGENIA. Bleeding risk is increased prior to platelet engraftment and may continue after engraftment in patients with prolonged thrombocytopenia. Two patients (
Patients should be made aware of the risk of bleeding until platelet recovery has been achieved. Monitor patients for thrombocytopenia and bleeding according to standard guidelines. Conduct frequent platelet counts until platelet engraftment and platelet recovery are achieved. Perform blood cell count determination and other appropriate testing whenever clinical symptoms suggestive of bleeding arise.
Neutrophil Engraftment Failure
There is a potential risk of neutrophil engraftment failure after treatment with LYFGENIA. Neutrophil engraftment failure is defined as failure to achieve three consecutive absolute neutrophil counts (ANC) ≥ 0.5 × 109 cells/L obtained on different days by Day 43 after infusion of LYFGENIA. Monitor neutrophil counts until engraftment has been achieved. If neutrophil engraftment failure occurs in a patient treated with LYFGENIA, provide rescue treatment with the back-up collection of CD34+ cells.
Insertional Oncogenesis
There is a potential risk of lentiviral vector-mediated insertional oncogenesis after treatment with LYFGENIA.
Hypersensitivity Reactions
Allergic reactions may occur with the infusion of LYFGENIA. The dimethyl sulfoxide (DMSO) or dextran 40 in LYFGENIA may cause hypersensitivity reactions, including anaphylaxis.
Anti-retroviral Use
Patients should not take prophylactic HIV anti-retroviral medications for at least one month prior to mobilization and until all cycles of apheresis are completed. There are some long-acting anti-retroviral medications that may require a longer duration of discontinuation for elimination of the medication. If a patient is taking anti-retrovirals for HIV prophylaxis, confirm a negative test for HIV before beginning mobilization and apheresis of CD34+ cells.
Hydroxyurea Use
Patients should not take hydroxyurea for at least 2 months prior to mobilization and until all cycles of apheresis are completed. If hydroxyurea is administered between mobilization and conditioning, discontinue 2 days prior to initiation of conditioning.
Iron Chelation
Drug-drug interactions between iron chelators and the mobilization process and myeloablative conditioning agent must be considered. Iron chelators should be discontinued at least 7 days prior to initiation of mobilization or conditioning. Do not administer myelosuppressive iron chelators (e.g., deferiprone) for 6 months post-treatment with LYFGENIA. Non-myelosuppressive iron chelation should be restarted no sooner than 3 months after LYFGENIA infusion. Phlebotomy can be used in lieu of iron chelation, when appropriate.
Interference with PCR-based Testing
Patients who have received LYFGENIA are likely to test positive by polymerase chain reaction (PCR) assays for HIV due to integrated BB305 LVV proviral DNA, resulting in a possible false-positive PCR assay test result for HIV. Therefore, patients who have received LYFGENIA should not be screened for HIV infection using a PCR-based assay.
Adverse Reactions
The most common adverse reactions ≥ Grade 3 (incidence ≥
Three patients died during LYFGENIA clinical trials; one from sudden cardiac death due to underlying disease and two from acute myeloid leukemia who were treated with an earlier version of LYFGENIA using a different manufacturing process and transplant procedure (Study 1, Group A).
Pregnancy/Lactation
Advise patients of the risks associated with myeloablative conditioning agents, including on pregnancy and fertility.
LYFGENIA should not be administered to women who are pregnant, and pregnancy after LYFGENIA infusion should be discussed with the treating physician.
LYFGENIA is not recommended for women who are breastfeeding, and breastfeeding after LYFGENIA infusion should be discussed with the treating physician.
Females and Males of Reproductive Potential
A negative serum pregnancy test must be confirmed prior to the start of mobilization and re-confirmed prior to conditioning procedures and before LYFGENIA administration.
Women of childbearing potential and men capable of fathering a child should use an effective method of contraception (intra-uterine device or combination of hormonal and barrier contraception) from start of mobilization through at least 6 months after administration of LYFGENIA.
Advise patients of the options for fertility preservation.
Please see full Prescribing Information for LYFGENIA including Boxed WARNING and Medication Guide.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days.
Founded in 2010, bluebird has been setting the standard for gene therapy for more than a decade—first as a scientific pioneer and now as a commercial leader. bluebird has an unrivaled track record in bringing the promise of gene therapy out of clinical studies and into the real-world setting, having secured FDA approvals for three therapies in under two years. Today, we are proving and scaling the commercial model for gene therapy and delivering innovative solutions for access to patients, providers, and payers.
With a dedicated focus on severe genetic diseases, bluebird has the largest and deepest ex-vivo gene therapy data set in the field, with industry-leading programs for sickle cell disease, β-thalassemia and cerebral adrenoleukodystrophy. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward.
bluebird continues to forge new paths as a standalone commercial gene therapy company, combining our real-world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements, such as statements regarding bluebird’s ability to ensure timely, equitable access to gene therapy; and the potential impact of the CGT Access Model on the coverage landscape for LYFGENIA. Such forward-looking statements are based on historical performance and current expectations and projections about bluebird’s future goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond bluebird’s control and could cause bluebird’s future goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect bluebird’s business, particularly those identified in the risk factors discussion in bluebird bio’s Annual Report on Form 10-K for the year ended December 31, 2023, as updated by its subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. These risks and uncertainties include, but are not limited to: delays and challenges in bluebird’s commercialization and manufacturing of its products; the internal and external costs required for bluebird’s ongoing and planned activities, and the resulting impact on expense and use of cash, has been, and may in the future be, higher than expected which has caused bluebird, and may in the future cause bluebird to use cash more quickly than it expects or change or curtail some of its plans or both; substantial doubt exists regarding bluebird’s ability to continue as a going concern; bluebird’s expectations as to expenses, cash usage and cash needs may prove not to be correct for other reasons such as changes in plans or actual events being different than bluebird’s assumptions; the risk that the efficacy and safety results from bluebird’s prior and ongoing clinical trials will not continue or be seen in the commercial context; the risk that QTCs experience delays in their ability to enroll or treat patients; the risk that bluebird experiences delays in establishing operational readiness across its supply chain; the risk that there is not sufficient patient demand or payer reimbursement to support continued commercialization of bluebird’s therapies; the risk of insertional oncogenic or other safety events associated with lentiviral vector, drug product, or myeloablation, including the risk of hematologic malignancy; and the risk that bluebird’s therapies will not be successfully commercialized. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, bluebird bio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.
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Investors:
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coleary@bluebirdbio.com
Media:
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jess.rowlands@bluebirdbio.com
Source: bluebird bio, Inc.
FAQ
When is the deadline for states to opt into bluebird bio's (BLUE) LYFGENIA coverage program?
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