The Diagnostics Pipeline: First FDA Approval of CRISPR Therapy Possible This Year
The agency recently announced it will decide in December whether to grant clearance for a CRISPR therapy for treating sickle cell disease.
Developers of CRISPR gene-editing therapies have made rapid progress and are now getting closer to regulatory approval. The FDA could decide as soon as December whether to grant its first clearance for a CRISPR therapy to Vertex Pharmaceuticals and CRISPR Therapeutics’ exa-cel for treating sickle cell disease and transfusion-dependent beta thalassemia.1 Meanwhile, the agency has established a new super office to handle the flow of future approval requests for novel gene and cell therapies.
The Promise of CRISPR Technology
Unlike therapies that correct defects in genes by adding functional copies of modified genes, clustered regularly interspaced short palindromic repeats (CRISPR) gene editing is capable of directly targeting and modifying specific DNA sequences.2 The technology uses a guide RNA that can steer the Cas9 enzyme to edit a targeted DNA sequence, like a pair of molecular scissors, to produce the desired change. CRISPR is also less expensive and thus more accessible than other gene therapy methods. As a result, there has been a boom in the development of CRISPR applications.
In addition to the ethics of its methods, one of the most controversial aspects of CRISPR gene editing technology is over who invented it. The major claimant is the Broad Institute, a research organization affiliated with Harvard University and the Massachusetts Institute of Technology (MIT). Rival claims for CRISPR invention come from biochemist Jennifer Doudna, PhD, and her colleagues from the University of California.3
In February 2022, the Broad Institute scored a major legal victory when the US Patent and Trademark Office ruled that it could keep its patents for CRISPR-Cas9 editing in eukaryotes, or organisms consisting of cells whose genetic material is DNA in the form of chromosomes contained within a distinct nucleus.4
Cell and Gene Products in the FDA Pipeline
The FDA has approved 32 different cell and gene therapies as of June 30,5 with the first approval granted in 2010 for a prostate cancer immunotherapy.6 Applications have been steadily growing, with the FDA’s Center for Biologics Evaluation and Research (CBER) now receiving over 200 investigational new drug (IND) applications per year, double the number from 2017, according to agency budgetary figures.7
The FDA Reorganizes
In preparation for the expected influx of new applications, CBER announced on March 15 that it has established an Office of Therapeutic Products (OTP) “to address the substantial growth in the development of innovative, novel products and the ever-changing public health landscape.” The new super office—the third created by the FDA—consists of six sub-offices, 14 divisions, and 33 branches. Among the sub-offices is the Office of Gene Therapy Chemistry, Manufacturing and Controls (CMC) to oversee the regulation of cell and gene therapy products.
The agency characterizes the restructuring as a move to “improve discipline alignment, increase review capacity, and enhance expertise in cell and gene therapies.” The FDA will also add review staff and supervisory positions to eliminate regulatory logjams, deal with the added workload, and speed up the approval process.8
Here are the key new laboratory testing and diagnostic products clearances announced from mid-May through mid-June 2023:
New FDA Approvals & Emergency Use Authorizations (EUAs)
|Cue Health||Cue COVID-19 Molecular Test, first at-home OTC test for COVID-19 to be granted De Novo authorization and first at-home test authorized using a traditional premarket review pathway for any respiratory illness|
|Foundation Medicine||Clearance for FoundationOne® Liquid CDx assay as companion diagnostic to Pfizer’s BRAFTOVI® (encorafenib) in combination with cetuximab for patients with metastatic colorectal cancer to identify BRAF V600E alteration|
|Acutis Diagnostics||EUA for SARS-CoV-2 Acutis Multiplex Assay|
|BioTeke USA||EUA for BIO-SELF™ COVID-19 Antigen Home Test home-use rapid antigen test for COVID-19|
|Hologic||510(k) clearance for a multiplex respiratory panel for SARS-CoV-2, influenza A and B|
|Thermo Fisher Scientific||Breakthrough device designation for B·R·A·H·M·S PlGF plus KRYPTOR and B·R·A·H·M·S sFlt-1 KRYPTOR immunoassays for preeclampsia run on firm’s B·R·A·H·M·S KRYPTOR compact PLUS clinical chemistry analyzer|
|Nano-Ditech||EUA for Nano-Check™ COVID-19 Antigen At-Home Test lateral flow assay|
|Becton Dickinson||510(k) clearance for BD Kiestra™ imaging application AI test for methicillin-resistant Staphylococcus aureus (MRSA) bacterial growth|
|Singlera Genomics||Breakthrough device designation for PDACatch assay, a DNA methylation-based liquid biopsy test for detection of pancreatic adenocarcinoma in high-risk individuals|
|BioMérieux||CLIA waiver for BIOFIRE® SPOTFIRE® Respiratory (R) Panel Mini for SARS-CoV-2, influenza A and B, respiratory syncytial virus, and rhinovirus|
New CE Marks & Global Certifications
Notable European CE certifications announced during the period:
New Approvals in Europe
|Euroimmun (subsidiary of Revvity)||UNIQO 160 fully automated indirect immunofluorescence test benchtop instrument|
|Seegene||30 assays, including eight for gastrointestinal infections, seven for women’s diseases, five for respiratory diseases, four for tuberculosis, three for meningitis, two for human papillomavirus (HPV), and one for drug resistance|
|Cepheid (subsidiary of Danaher)||Xpert® NPM1 Mutation, RT-PCR assay for acute myeloid leukemia (AML) patients|
|Eurobio Scientific||Three unspecified PCR tests from firm’s EurobioPlex line|
|Regen Lab||REGENKIT® preparation of platelet-rich plasma (PRP)|
|Regen Lab||CELLULAR MATRIX fig®, a combination of PRP and hyaluronic acid (HA)|
|Regen Lab||Arthrovisc® comprised of syringes filled with HA|
|Invivoscribe||LeukoStrat® CDx FLT3 Mutation Assay for acute myelogenous leukemia|
|SphingoTec||sphingotest® penKid® assay for helping diagnose acute kidney injury|
Other international clearances announced during the period:
|Sysmex||Japan||PrismGuide™ IRD Panel System for identifying genes that cause inherited retinal dystrophy (IRD)|
|Eurobio Scientific||UK||Three unspecified PCR tests from firm’s EurobioPlex line|
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