Perspectives
Gene/Cell Therapy Quarterly Update: September 2023
Critical updates in an ever-changing environment
September 25, 2023This quarterly pipeline wrap-up provides a review of newly approved gene and cell therapies, new indications and news of note on gene and cell therapies drugs in the approval process. See other articles for updates on specialty drugs, biosimilar drugs and traditional drugs.
FDA approved Gene/Cell Therapy Products in the last 12 months
| Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | Month Approved | |
| valoctocogene roxaparvovec-rvox | Roctavian™ | BioMarin | Severe hemophilia A | IV | June 2023 | |
| delandistrogene moxeparvovec-rokl | Elevidys® | Sarepta Therapeutics/
Roche |
Duchenne muscular dystrophy (DMD)
(4-5 years of age) |
IV | June 2023 | |
| donislecel-jujn | Lantidra® | CellTrans Inc | Uncontrolled type 1 diabetes | Hepatic portal vein | June 2023 | |
| beremagene geperpavec-svdt | Vyjuvek® | Krystal Biotech | Dystrophic epidermolysis bullosa (DEB) | Topical | May 2023 | |
| omidubicel-onlv | Omisirge® | Gamida Cell | Blood cancers in need of allogeneic hematopoietic stem cell transplant (HSCT) | Cell therapy | April 2023 |
Gene and Cell Therapy Pipeline (Submitted to the FDA)
| Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | PDUFA Date | Estimated Prevalence in the US |
| debamestrocel
|
NurOwn® | BrainStorm Cell Therapeutics | Amyotrophic lateral sclerosis
|
Intrathecal | December 2023 | 25,000 |
| exagamglogene autotemcel | N/A | Vertex | Sickle cell disease (SCD) | IV | December 2023 | 20,000 |
| Transfusion dependent
beta-thalassemia (TDT) |
April 2024 | 1,500 | ||||
| lovotibeglogene autotemcel | N/A | bluebird bio | SCD | IV | December 2023 | 20,000 |
| lifileucel | N/A | Iovance | Unresectable or metastatic melanoma | CAR-T cell therapy | February 2024 | 180,000 |
| atidarsagene autotemcel | N/A | Orchard | Metachromatic leukodystrophy | IV | March 2024 | 2,500 |
| fidanacogene elaparvovec | N/A | Roche & Pfizer | Hemophilia B | IV | April 2024 | 3.9-6.5/million |
Extended Gene Therapy* 2023-2026 Pipeline
| Generic Name | Brand Name | Manufacturer | Indication(s) | Route of Administration | Anticipated Filing date+ | Estimated Prevalence in the U.S. |
| mametegragene autotemcel | N/A | Rocket | Severe leukocyte adhesion deficiency type 1 (LAD-1) | IV | Submitted | 25-50/year |
| eladocagene exuparvovec | Upstaza™ | PTC Therapeutics | Aromatic L-amino acid decarboxylase (AADC) deficiency | Intracerebral | 3Q2023 | 300 in world |
| EB101
(prademagene zamikeracel) |
N/A | Abeona | Recessive DEB | Topical/surgical – One time treatment | 2H2023 | 400 |
| mozafancogene autotemcel | N/A | Rocket | Fanconi Anemia | IV | 4Q2023 | 6.25/million |
| RGX-121 | N/A | Regenxbio | Hunter’s syndrome | Intracisternal | 1H2024 | <1/million |
| UX-701 | N/A | Ultragenyx | Wilson disease | IV | 2H2024 | 9,000 |
| SB-525
(giroctocogene fitelparvovec) |
N/A | Sangamo/
Pfizer |
Severe hemophilia A | IV | 3Q2024 | 8/100,000 males |
| fordadistrogene movaparvovec | N/A | Pfizer | DMD | IV | 4Q2024 | 9,000-12,000 |
| botaretigene sparoparvovec | N/A | J & J and
MeiraGTx |
X-linked retinitis pigmentosa | Subretinal | 2H2024 | 20,000 in US and Europe |
| FLT180a
(verbrinacogene setparvovec) |
N/A | Freeline Therapeutics | Moderate to severe hemophilia B | IV | 2H2024 | 1.5/100,000 males |
| pariglasgene brecaparvovec | N/A | Ultragenyx | Glycogen storage disease type 1a | IV | 2H2024 | 8/million |
| lenadogene nolparvovec
(GS-010) |
Lumevoq™ | GenSight Biologics | Leber Hereditary Optic Neuropathy | Intravitreal injection | 1H2025 | 6.5/million |
| SRP-9003
(bidridstrogene xeboparvovec) |
N/A | Sarepta | Limb girdle muscular dystrophy type 2E | IV | 1H2025 | 3.5/million |
| AMT-130 | N/A | uniQure | Huntington’s disease | Intracerebral | 2H2025 | 15.2/million |
| AGTC-501 (laruparetigene zosaparvovec) | N/A | AGTC | X-linked retinitis pigmentosa | Subretinal | 2H2025 | 20,000 in US and Europe |
| OTL-201 | N/A | Orchard | MPS IIIA | IV | 2026 | 1,000 |
| PBGM-01 | N/A | Passage Bio | GM1 gangliosidosis | Intrathecal | 2026 | TBD |
| MYR101 | N/A | Myrtelle | Canavan disease | Intracerebro-ventricular | 2026 | TBD |
| RP-A501 | N/A | Rocket | Danon Disease | IV | 2026 | TBD |
| RGX-314
|
N/A | Regenxbio | Wet age-related macular degeneration | Subretinal | 2026 | 1.1 million |
IV=intravenous; CAR=chimeric antigen receptor; TBD=to be determined
*Gene Therapy: One-time administration with curative intent and anticipated WAC of > $750,000
+Estimates based on clinical evidence available at the time of publication
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