Since the new FDA Commissioner Dr. Scott Gottlieb was sworn in on May 11, 2017, the traditionally cumbersome regulatory body has shown an unprecedented embracement for novelty in treatments and beyond.
Overarching guidelines:
- Digital Health Innovation Action Plan – July 2017
- Breakthrough Devices Program Guidance – draft issued October 2017, finalized December 2018
- FDA Unveils 6 New Draft Guidances on Gene Therapy – July 2018
Here is a roundup of those approvals
Novartis, Kymriah, first gene (transfer) therapy (first CAR-T) available in the United States
- August 30, 2017
- Biologics License Application (BLA) submitted on February 2, 2017
- Approval basis: the safety and efficacy of Kymriah were demonstrated in one multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was 83 percent. (FDA statement)
Pear Therapeutics, the Reset app, first mobile application to treat substance use disorders (SUD)
- September 14, 2017
- de novo premarket review pathway
- Technology: the device delivers cognitive behavioral therapy to patients to teach the user skills that aid in the treatment of SUD and are intended to increase abstinence from substance abuse and increase retention in outpatient therapy programs. The system is intended to be used in conjunction with outpatient therapy and in addition to a contingency management system, a widely-used program for treating SUD that uses a series of incentives to reward patients for adherence to their treatment program.
- Approval basis: a multi-site, unblinded 12-week clinical trial of 399 patients who received either standard treatment or standard treatment with the addition of a desktop-based version of Reset which could be accessed at the clinic or at home. The data showed a statistically significant increase in adherence to abstinence for the patients with alcohol, cocaine, marijuana and stimulant SUD in those who used Reset, 40.3 percent, compared to the patients who did not, 17.6 percent. The clinical trial did not demonstrate the effectiveness of using the Reset device in patients reporting opioids as their substance of abuse. (FDA statement)
Memorial Sloan Kettering Cancer Center, IMPACT test, first tumor-profiling laboratory-developed test to receive authorization through the FDA
- November 15, 2017
- de novo premarket review pathway
- Technology: the IMPACT test uses next-generation sequencing (NGS) to rapidly identify the presence of mutations in 468 unique genes, as well as other molecular changes in the genomic makeup of a person’s tumor.
- Approval basis: results indicated that the assay is highly accurate (greater than 99 percent) and capable of detecting a mutation at a frequency of approximately 5 percent (range of 2-5 percent). Additionally, detection of certain molecular changes (microsatellite instability) using the IMPACT test was concordant more than 92 percent of the time across multiple cancer types in 175 cases, when compared to traditional methods of detection.
- Along with this authorization, the FDA is also establishing a Class II regulatory pathway for the review of other NGS-based tumor profiling tests for use in patients diagnosed with cancer. Class II designation allows these types of tests to be eligible to use the FDA’s 510(k) clearance process, either by submitting the application. (FDA statements)
Proteus Digital Health x Otsuka Pharma, Abilify MyCite, first drug in the U.S. with a digital ingestion tracking system
- November 13, 2017
- New Drug Application (NDA) dated and received June 26, 2015
- Technology: the system works by sending a message from the pill’s sensor to a wearable patch. The patch transmits the information to a mobile application so that patients can track the ingestion of the medication on their smart phone. Patients can also permit their caregivers and physician to access the information through a web-based portal.
- Approval basis: Abilify was first approved by the FDA in 2002 to treat schizophrenia. The ingestible sensor used in Abilify MyCite was first permitted for marketing by the FDA in 2012. (FDA statement)
Spark Therapeutics, Luxturna, first (directly administered) gene therapy approved in the U.S. to target a disease caused by mutations in a specific gene
- December 19, 2017
- Biologics License Application (BLA) dated April 26, 2017, and received May 16, 2017
- Approval basis: The efficacy of LUXTURNA in the Phase 3 study was established based on the multi-luminance mobility test (MLMT) score change from baseline to one year. LUXTURNA Phase 3 clinical study results showed a statistically significant difference between the intervention group (n=21) and control participants (n=10) at one year in median bilateral MLMT score change (intervention minus control group difference of 2; p=0.001) and median first-treated eye MLMT score change (intervention minus control group difference of 2; p=0.003). (Spark press release)
- FDA statement
Loxo Oncology, Vitrakvi, first time that an entirely new treatment has received a tumor-agnostic indication at its initial approval
- November 26, 2018
- covered in a previous blog: Loxo and Pan-Cancer Treatments
Apple, the ECG app (with Apple Watch), first FDA clearance for retail ECG watch technology
- September 11, 2018
- De Novo clearance from the Center for Devices and Radiological Health, classified as Class II under the generic name electrocardiograph software for over-the-counter use
- Technology: the ECG app determines the presence of atrial fibrillation (AFib) or sinus rhythm on a classifiable waveform
- FDA statement
- FDA letter to Apple
- covered in a previous blog: New Era of Medical Grade Consumer Device
- January 17, 2019 update: Alphabet’s life sciences unit Verily received 510(k) clearance from FDA as a Class II medical device for its on-demand ECG feature
