CRISPR is a powerful gene-editing tool that enables targeted therapeutic gene editing, with clinical applications for improving treatment of inherited and/or rare genetic diseases, viral infections, and arresting the progression of cancer. CRISPR gene-editing technology has the potential to revolutionize the treatment of rare genetic disorders, with the first product poised for FDA approval in…
The dominant amyloid hypothesis of AD has translated into an armada of anti-amyloid biologics in the near-term pipeline—led by Eisai and Biogen’s lecanemab. Last month, lecanemab showed a slowing in the rate of cognitive decline by 27% over 18 months versus placebo. Lecanemab also possesses a relatively improved safety profile among the beta-amyloid mAbs, as…
Traditional clinical trials are typically conducted at central locations or sites that patients must travel to in order to be evaluated and treated by trial investigators. These are referred to as centralized clinical trials. In contrast, a decentralized clinical trial (DCT) has no set location for patients to report to. Rather, clinical trial activities are…
On September 30, 2022, President Biden signed into law the reauthorization of the Prescription Drug User Fee Act (PDUFA VII), which will be in place for the next 5 years. Despite extensive bipartisan efforts to include reforms of the accelerated approval (AA) pathway as so-called “policy riders” in the bill, ultimately a “practically clean” version…
Real-world data (RWD) can be used to create historical control groups for clinical trials in rare diseases where a randomized controlled trial (RCT) is not feasible. But what happens when the US Food and Drug Administration (FDA) doesn’t accept it? Since passage of the 21st Century Cures Act in 2016, FDA has promoted the use…
The United States is facing an avalanche of Alzheimer’s disease (AD). An estimated 12.7 million Americans over the age of 65 are projected to suffer from AD dementia by 2050,1 and yet, despite more than 30 years of intensive research, we have yet to develop a drug that provides a clinically meaningful slowing in cognitive…
In Part 1 of our blog series on the saga of PI3K inhibitors, we reviewed the FDA’s recent Oncologic Drugs Advisory Committee (ODAC) meeting on April 21, 2022, to discuss the agency’s concerns about PI3K inhibitors (PI3Kis). The panel voted resoundingly (16 yes votes; 1 abstention) that future approvals of PI3Kis should be supported by…
The FDA’s Oncology Division has recently taken a hard stance on PI3K inhibitors, a novel class of drugs that inhibit various isoforms of phosphatidylinositol 3-kinase (PI3K) and are approved for treating blood cancers. Recent actions by the FDA, including withdrawing some approved indications, indicate that they will likely be seeking a higher bar for new…
By Jackie Orabone, PhD and Angela Corona, PhD The accelerated approval (AA) pathway was introduced in 1992 (in response to the AIDS epidemic) to shorten the FDA approval process for drugs to treat serious or life-threatening diseases or rare diseases where there is a high unmet medical need. AA allows for drugs to be approved…
The FDA has developed several mechanisms to speed drugs to market when a compelling medical need exists including Accelerated Approval (AA), Priority Review, Fast Track, and Breakthrough Therapy Designation. Accelerated Approval is an important regulatory pathway that provides patients with earlier access to treatments for serious medical conditions when there is an unmet medical need….
