When it comes to cell and gene therapy revenues, it’s feast or famine. The feast side is well covered in this A-List, which ranks the top 10 therapies according to sales figures from 2023. The famine side begins almost exactly where this A-List ends. Future A-Lists are bound to push famine farther into the background as additional gene therapies are approved. For example, according to the Alliance for Regenerative Medicine, up to 17 cell and gene therapies could be approved this year alone in the U.S. and Europe (up from the 5 gene therapies that were approved in 2023).

Gene therapies that employ in vivo gene editing technology look especially promising now that the first such therapy, CRISPR-based Casgevy (exagamglogene autotemcel), has secured FDA approval. (In December 2023, it was approved for sickle cell disease; in January 2024, it was approved for beta-thalassemia.) According to Goldman Sachs analysts, peak-year sales for Casgevy are projected to reach $3.9 billion.

Another encouraging development is the success of Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy indicated for Duchenne muscular dystrophy. It generated just over $200 million in net product revenue last year, its first year on the market, despite being available to patients only since the summer.

During Sarepta’s Q4 2023 quarterly earnings call, Dallan Murray, the company’s executive vice president and chief customer officer, told analysts, “The success of Elevidys shows that gene therapy can be commercially viable, providing hope for those patients with Duchenne, and for all those with genetic conditions and unmet needs.” He added that Elevidys performed the best of the six gene therapies launched since mid-2022.

Of these six, the second-best performer in terms of revenues was Krystal Biotech’s Vyjuvek (beremagene geperpavec-svdt), which made $50.7 million in 2023—too little to be among the top 10 therapies in this A-List, which ranks cell and gene therapies by 2023 revenues as disclosed by the sponsoring companies in regulatory filings, press releases, financial statements, or quarterly earnings calls.

Farther beyond the top 10, the sales figures drop off considerably. Bluebird Bio’s Zynteglo (betibeglogene autotemcel, “beti-cel”) made $16.7 million; Bluebird’s Skysona (elivaldogene autotemcel, “eli-cel”), $12.4 million; BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox, “val-rox”), $3.5 million. Even lower was a cell therapy launched last year, Gamida Cell’s cell therapy Omisirge (omidubicel-onlv), which finished 2023 generating just $1.784 million.

Of the 12 gene therapies on the market in the United States as of December 31, 2023, only 7 had sales figures publicly available. Of the other five, sales figures for four of them were not disclosed by their sponsors: Ferring Pharmaceuticals’ Adstiladrin (nadofaragene firadenovec-vncg); CSL Behring’s Hemgenix (etranacogene dezaparvovec-drlb); BioVex’s (Amgen’s) Imlygic (talimogene laherparepvec); and PTC Therapeutics’ Upstaza (eladocagene exuparvovec). The fifth gene therapy, Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel, “lovo-cel”) had yet to generate revenue last year, though Bluebird said on March 26 that it expects to recognize revenue from its first infusion of Lyfgenia in the third quarter of this year.

This A-List does not include antisense oligonucleotides (ASOs) even though therapies of this class are sometimes referred to as gene therapies. Our rationale is that ASOs modulate RNA instead of manipulating or inserting genetic DNA. Another reason: ASOs are not included in the FDA’s list of Approved Celllular and Gene Therapy Products. However, if this A-List had included ASOs, we would have listed two treatments from Sarepta Therapeutics for Duchenne muscular dystrophy. Exondys 51 (eteplirsen) and Amondys 45 (casimersen) are for patients who have mutations amenable to exon 51 skipping and exon 45 skipping, respectively. The 2023 sales figures for these therapies are $540.576 million and $273.755 million, respectively.

 

1. Yescarta (axicabtagene ciloleucel)

2023 Sales: $1.498 billion

Sponsor(s): Kite, a Gilead Company

Type: CD19-directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adults with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy. Also, treatment of adults with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and diffuse large B-cell lymphoma arising from follicular lymphoma.

Initial FDA Approval Date: October 18, 2017

 

2. Zolgensma (onasemnogene abeparvovec-xioi)

2023 Sales: $1.214 billion

Sponsor(s): Novartisa

Type: Adeno-associated virus vector–based gene therapy

Indication(s): Treatment of pediatric patients less than two years of age with spinal muscular atrophy with biallelic mutations in the survival motor neuron 1 gene.

Initial FDA Approval Date: May 24, 2019

 

3. Kymriah (tisagenlecleucel)

2023 Sales: $508 million

Sponsor(s): Novartis

Type: CD19-directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia that is refractory or in second or later relapse; adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, high grade B-cell lymphoma and diffuse large B-cell lymphoma arising from follicular lymphoma; and adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy.

FDA Approval Date: August 30, 2017

 

4. Carvykti (ciltacabtagene autoleucel)

2023 Sales: $500 million

Sponsor(s): Janssen Biotech (Johnson & Johnson) and Legend Biotech

Type: B-cell maturation antigen–directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Initial FDA Approval Date: February 28, 2022

 

5. Abecma (idecabtagene vicleucel)

2023 Sales: $472 million

Sponsor(s): Bristol Myers Squibb and 2seventy bio

Type: B-cell maturation antigen–directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adult patients with relapsed or refractory multiple myeloma after two or more prior lines of therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

Initial FDA Approval Date: March 26, 2021

 

6. Tecartus (brexucabtagene autoleucel)

2023 Sales: $370 million

Sponsor(s): Kite, a Gilead Company

Type: CD19-directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adults with relapsed or refractory mantle cell lymphoma. Also, treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

Initial FDA Approval Date: July 24, 2020 (accelerated approval)

 

7. Breyanzi (lisocabtagene maraleucel)

2023 Sales: $364 million

Sponsor(s): Bristol Myers Squibb

Type: CD19-directed genetically modified autologous T-cell immunotherapy

Indication(s): Treatment of adults with large B-cell lymphoma, including diffuse large B-cell lymphoma not otherwise specified (including diffuse large B-cell lymphoma arising from indolent lymphoma), high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B who have refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy, or refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplantation due to comorbidities or age, or relapsed or refractory disease after two or more lines of systemic therapy. Also, treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma who have received at least two prior lines of therapy, including a Bruton’s tyrosine kinase inhibitor and a B-cell lymphoma 2 inhibitor.

Initial FDA Approval Date: February 5, 2021 (accelerated approval)

 

8. Elevidys (delandistrogene moxeparvovec-rokl)

2023 Sales: $200.356 million

Sponsor(s): Sarepta Therapeutics

Type: Adeno-associated virus vector–based gene therapy

Indication(s): Treatment of ambulatory pediatric patients aged four through five years with Duchenne muscular dystrophy with a confirmed mutation in the Duchenne muscular dystrophy gene.

Initial FDA Approval Date: June 22, 2023 (accelerated approval)

 

9. MACI (autologous cultured chondrocytes on porcine collagen membrane)

2023 Sales: $164.8 million

Sponsor(s): Vericel

Type: Autologous cellularized scaffold product

Indication(s): Repair of symptomatic, single, or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults.

Initial FDA Approval Date: December 13, 2016

 

10. Luxturna (voretigene neparvovec-rzyl)

2023 Sales: Approximately CHF 46 million (about $51 million)b

Sponsor(s): Spark Therapeutics (Roche)

Type: Adeno-associated virus vector–based gene therapy

Indication(s): Treatment of patients with confirmed biallelic RPE65 mutation–associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).

Initial FDA Approval Date: December 18, 2017

 

Footnotes

a Novartis is the successor to AveXis, which successfully completed the development of Zolgensma in 2019 by receiving FDA approval for the gene therapy. In 2014, AveXis licensed from Regenxbio the AAV9 vector used in the Phase I SMA clinical trial at Nationwide Children’s Hospital. Regenxbio licensed exclusive rights to key intellectual property covering novel recombinant AAV vectors discovered at University of Pennsylvania in the laboratory of James M. Wilson, MD, PhD.

b Roche has not furnished a 2023 Luxturna sales figure, instead including those sales within the “others” portion of its ophthalmology sales listed in its 2023 annual report, CHF 48 million (about $53 million). That figure is the same as the “others” figure listed in 2022, when the company disclosed CHF 46 million in Luxturna sales.

Source:
https://www.genengnews.com/a-lists/top-10-best-selling-cell-and-gene-therapies/
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