RegulationBreakthrough Therapy (US), Accelerated Approval (US), Orphan Drug (US), Rare Pediatric Disease (US), Orphan Drug (EU), PRIME (EU), Accelerated assessment (EU)

Gene Sequence

Sequence Code 538965991

Source: *****

Clinical Trials associated with Elivaldogene Autotemcel (Bluebird Bio)

NCT03852498 / CompletedPhase 3

A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)

The purpose of this study is to evaluate the efficacy and safety of Lenti-D Drug Product (also known as elivaldogene autotemcel or Skysona, hereafter referred to as eli-cel) after myeloablative conditioning with busulfan and fludarabine in participants with CALD. A participant's blood stem cells will be collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells will be transplanted back into the participant following myeloablative conditioning. Enrollment and treatment in Study ALD-104 have been completed and further enrollment in this study is not expected, although participants follow-up remains ongoing in the long-term follow-up Study LTF-304 (NCT02698579).

Start Date24 Jan 2019

Sponsor / Collaborator

bluebird bio, Inc.

NCT02698579 / Active, not recruitingNot Applicable

Long-term Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

This is a multi-center, long-term safety and efficacy follow-up study for participants with cerebral adrenoleukodystrophy (CALD) who have received Lenti-D Drug Product (eli-cel) in a parent clinical study.
After completing a parent clinical study (approximately 2 years), eligible participants will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in this study.

Start Date22 Jan 2016

Sponsor / Collaborator

bluebird bio, Inc.

NCT01896102 / CompletedPhase 2/3

A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)

This trial assessed the efficacy and safety of autologous cluster of differentiation 34 (CD34+) hematopoietic stem cells, transduced ex-vivo with Lenti-D lentiviral vector (also called elivaldogene autotemcel or eli-cel), for the treatment of cerebral adrenoleukodystrophy (CALD). A participant's blood stem cells were collected and modified (transduced) using the Lenti-D lentiviral vector encoding human adrenoleukodystrophy protein. After modification (transduction) with the Lenti-D lentiviral vector, the cells were transplanted back into the participant following myeloablative conditioning. Participants in this study will be continuously followed in study LTF-304.

Start Date21 Aug 2013

Sponsor / Collaborator

bluebird bio, Inc.

100 Clinical Results associated with Elivaldogene Autotemcel (Bluebird Bio)

100 Translational Medicine associated with Elivaldogene Autotemcel (Bluebird Bio)

100 Patents (Medical) associated with Elivaldogene Autotemcel (Bluebird Bio)

Literatures (Medical) associated with Elivaldogene Autotemcel (Bluebird Bio)

01 Nov 2021·Molecular diagnosis & therapyQ4 · MEDICINE

Elivaldogene Autotemcel: First Approval

Q4 · MEDICINE

Review

Author: Keam, Susan J

Elivaldogene autotemcel (SKYSONA™, eli-cel; Lenti-D™ gene therapy) is a gene therapy that has been developed by bluebird bio for the treatment of cerebral adrenoleukodystrophy (CALD), a rare, X-linked genetic disease that mainly affects the nervous system and adrenal glands. In July 2021, elivaldogene autotemcel received its first approval (in the EU) for the treatment of for the treatment of early CALD in patients < 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling haematopoietic stem cell (HSC) donor is not available. This article summarizes the milestones in the development of elivaldogene autotemcel leading to this first approval.

01 Jun 2006·Deutsche medizinische Wochenschrift (1946)Q4 · MEDICINE

Q4 · MEDICINE

Article

Author: Bartl, R ; Hammerschmidt, T ; Götte, S ; Hadji, P

BACKGROUND AND OBJECTIVE:

Bisphosphonates provide efficacious treatment for osteoporosis. However, side effects often lead to patients discontinuing this treatment. This study analyses differences in adherence (including acceptance, persistence and compliance) between daily (ALD-D) and weekly (ALD-W) administration of alendronate among German patients with osteoporosis.

METHODS:

Prescription claims data of two random groups of subjects (144 patients each) taking ALD-D and ALD-W, respectively, were observed for 12 months after starting the given prescription. Termination was defined as the moment when the last prescription had been used up. The percentage of patients continuing the drug treatment after the first prescription was used as a measure for acceptance. Compliance was measured by the medication possession ratio (MPR), namely the percentage of days on which the patient was supplied with the medication. An MPR > 80% is therapeutically relevant because the risk of fractures is significantly reduced.

RESULTS:

31.3% (ALD-W) vs. 45.8% (ALD-D) of patients discontinued therapy after one prescription. 53.5% (ALD-W) vs. 72.2% (ALD-D) of patients discontinued therapy throughout the year. The proportion of those who discontinued the treatment was significantly higher with daily administration (p=0.0035). Mean time until discontinuation was 220 days (ALD-W) vs. 169 days (ALD-D). Mean compliance among any patients was 51.7% (ALD-W) vs. 37.7% (ALD-D); Only 30.6% (ALD-W) vs. 19.2% (ALD-D) (p=0,0295) of patients reached a therapeutically relevant compliance level.

CONCLUSIONS:

A large proportion of patients discontinued treatment with bisphosphonates, a majority of discontinuing patients not even refilling their first prescription. Adherence, although enhanced by less frequent dosing, was suboptimal in all of its aspects, i.e. acceptance, persistence and compliance. There is a need for treatment strategies to increase adherence.

01 Jan 1983·Journal of the American Academy of DermatologyQ1 · MEDICINE

Sensitivity to α-amylcinnamic aldehyde and α-amylcinnamic alcohol

Q1 · MEDICINE

Article

Author: Jere D. Guin ; Philip Haffley

Sensitivity to alpha-amylcinnamic aldehyde (alpha-AcAld) is apparently uncommon, but, like allergy to alpha-amylcinnamic alcohol (alpha-AcAlc), it often accompanies allergy to the perfume in Mycolog cream. Although alpha-AcAlc is a known ingredient, alpha-AcAld is not. However, gas-liquid chromatographic analysis shows alpha-AcAld to be present. Of fourteen persons sensitive to either chemical, ten reacted to both. Of these, one man and three women were markedly sensitive, and all three women had chronic recalcitrant vulvar eczema. That condition might have been the cause as well as the result of sensitization, but reexposure to a suspected product reproduced the eruption in both persons tested. Its use with other potent sensitizers, e.g., ethylenediamine, to treat irritations and chronic eczemas in an area of high absorption may partly explain development of allergy to a relatively weak sensitizer.

News (Medical) associated with Elivaldogene Autotemcel (Bluebird Bio)

10 May 2024

·www.biopharmadive.com

iTeos shares jump on TIGIT update; Acelyrin swaps CEOs

BioPharma Dive is testing out a new format rounding up smaller updates from around the industry. Have thoughts on what could make this type of story better? Drop us a line!Today, a brief rundown of news from iTeos Therapeutics, as well as notes on Gilead, Acelyrin and Bluebird bio that you may have missed yesterday:Shares in iTeos Therapeutics jumped by nearly 40% Friday morning after the biotechnology company gave an update on a clinical trial of a cancer drug combination its studying with GSK. Interim data surpassed iTeos pre-defined efficacy criteria for clinically relevant activity, the company said. The study is testing iTeos anti-TIGIT antibody together with GSKs PD-1 inhibitor Jemperli in first-line treatment of non-small cell lung cancer.Ned PagliaruloBluebird bio made national headlines in The New York Times this week with news it had collected the cells of the first patient set to undergo treatment with its sickle cell disease therapy Lyfgenia. In an earnings update Thursday, the company disclosed that, so far this year, 14 patients have started the treatment process for its other two marketed gene therapies 11 for Zynteglo, which treats severe beta thalassemia, and 3 for Skysona, approved for a rare brain disorder. Bluebird expects between 85 and 105 patient starts for its three therapies this year. Ned PagliaruloImmune drug developer Acelyrin said Thursday that founder Shao-Lee Lin has stepped down as CEO and will be replaced by current chief legal and administrative officer Mina Kim. The biotech raised $540 million in an initial public offering last year, but has lost much of its value after hitting a clinical trial setback in September. Ben FidlerGilead Sciences and partner Arcellx on Thursday afternoon disclosed the design of a Phase 3 trial they plan to run testing a CAR-T therapy in people with relapsed or refractory multiple myeloma. The therapy, dubbed anito-cel, targets a protein called BCMA, similar to the approved cell therapies Carvykti, from Johnson & Johnson, and Abecma, from Bristol Myers Squibb.Ned Pagliarulo '

Phase 3Cell TherapyExecutive ChangeImmunotherapyGene Therapy

09 May 2024

·endpts.com

Bluebird bio starts 15 patients on its three multimillion-dollar gene therapies

Bluebird bio’s stock $BLUE jumped over 20% on Thursday following its first-quarter earnings release and the announcement of 15 patients starting on its three approved gene therapies: Zynteglo, Skysona and Lyfgenia. The company wrote in its financial report that 15 patients had completed starts on its expensive gene therapies: 11 on Zynteglo for transfusion-dependent thalassemia, three on Skysona for cerebral adrenoleukodystrophy and one on Lyfgenia for sickle cell. Bluebird also expects 85 to 105 more patient starts across its portfolio this year. You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

Gene Therapy

09 May 2024

·firstwordpharma.com

Uptick in gene therapy uptake triggers stock bump for beleaguered bluebird

Bluebird bio’s three gene therapies may finally be gaining momentum. The biotech shared in its first-quarter earnings report that, so far this year, 15 new patients have started on one of the multimillion-dollar treatments – and it’s anticipating over 100 starts in total for 2024. Shares of bluebird jumped 15% on the news Thursday.With a market cap now hovering around $200 million, bluebird has struggled with safety concerns, submission delays, and the rollout of its gene therapy portfolio; at its peak in 2018, the biotech had $1.6 billion in cash on hand. For more analysis, see Vital Signs: The last flight of the bluebird. However, the company may have turned a corner this year. After scoring FDA approval in December for its one-time sickle-cell disease treatment Lyfgenia (lovotibeglogene autotemcel) – priced at $3.1 million – bluebird has since secured multiple outcomes-based agreements with commercial payers, and the US Centers for Medicare and Medicaid Services (CMS) has proposed to raise reimbursement rates for the therapy. One new patient has started Lyfgenia this year. Bluebird’s $2.8-million beta-thalassemia gene therapy, Zynteglo (betibeglogene autotemcel), saw 11 new patient starts, and Skysona (elivaldogene autotemcel), a $3-million treatment for cerebral adrenoleukodystrophy, had three. Approved in 2022, Zynteglo and Skysona carry middling price tags for gene replacement therapies – far less than that of Lenmeldy (atidarsagene autotemcel) from Orchard Therapeutics, the most expensive treatment at $4.25 million. For more, see Vital Signs: Shrinkflation hits the gene therapy sector.Bluebird bio reported net revenues of $18.6 million for the first quarter, a huge jump over its $2.4 million revenues for the year-ago period. While bluebird didn’t provide full-year revenue guidance, it expects between 85 to 105 new patient starts across its three gene therapies.

Gene TherapyDrug Approval

100 Deals associated with Elivaldogene Autotemcel (Bluebird Bio)

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Adrenoleukodystrophy	EU	bluebird bio, Inc.	16 Jul 2021
Adrenoleukodystrophy	IS	bluebird bio, Inc.	16 Jul 2021
Adrenoleukodystrophy	LI	bluebird bio, Inc.	16 Jul 2021
Adrenoleukodystrophy	NO	bluebird bio, Inc.	16 Jul 2021

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT01896102 (ALD-102, BusinessWire) Manual	Phase 2/3	Adrenoleukodystrophy	32	Elivaldogene Autotemcel	dsnlkgzeij(geqxiqjcxf) = mrzfixvugx btyiaqaenb (oxvrgqvaou ) View more	Positive	15 Mar 2021
NCT01896102 (2011-001953-10, EBMT2018)	Phase 2/3	Adrenoleukodystrophy ABCD1	21	Lenti-D gene therapy	lobyqpvojr(ocdlshctgr) = yuiwuhswiu gatxayqngg (ojndwbygou ) View more	Positive	24 Sep 2018
NCT01896102 (STARBEAM, Pubmed \| Br Dent J) Manual	Phase 2/3	Adrenoleukodystrophy	17	elivaldogene tavalentivec	ijeysrfalo(bmsimvwsli) = livsqerxzc auxtdpvufm (lsjghyvehh )	Positive	26 Oct 2017

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Elivaldogene Autotemcel (Bluebird Bio)

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