FDA approvals Archives - Medical Design Sourcing

Biotech bounces back at JPM 2024 on optimism, breakthroughs and calculated bets, but uncertainties persist

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At the dawn of 2024, there’s a sense of renewed optimism in the biotech sector despite a downturn that has lasted for more than two years. At the JP Morgan Health Care Conference, deal-making activity showed signs of strength. For instance, Merck agreed to acquire cancer drug developer Harpoon Therapeutics for roughly $680 million, highlighting continued interest in oncology cancer therapies. Meanwhile, Novartis announced plans to acquire Calypso Biotech, a deal involving an upfront payment of $250 million with potential milestones worth up to $175 million.

“I feel like in the last two or three weeks, we’ve almost made up for 50% of the deals that didn’t happen in 2023,” quipped Jen Nwankwo, CEO of 1910 Genetics, a company specializing in computational biology and automated laboratory technologies. Pointing to recent clinical successes and FDA approva…

Core trends in 2023 FDA drug approvals: Oncology, neurology and hematology dominate

2023 was a big year for hematology, neurology and oncology, with the medical specialties seeing the most FDA approvals. In terms of sponsors, Pfizer had the most approvals with six total, followed by UCB and Chiesi, each with three apiece.

When looking at commercial prospects, AstraZeneca’s respiratory syncytial virus antibody Beyfortus could be the biggest blockbuster from the 2023 cohort with expected peak sales of $1.9 billion. Alzheimer’s drug Leqembi could also be a major success, making up for the tepid demand for Aduhelm, which won conditional approval in 2021. While Aduhelm’s sales have sputtered, Leqembi could see peak revenue of $4.8 billion according to analysts.

Oncology continues to be a hot specialty with the most competition among the newly approved drugs, as the FDA continues to green light new entrants in crowded target classes such as PD-1 inhibitors.

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Roche enters red-hot metabolic disease market with Carmot Therapeutics acquisition

Swiss pharma giant Roche had a limited presence in metabolic disease, but the firm has agreed to take over obesity drug developer Carmot Therapeutics for $2.7 billion, rivaling the dominance of next-gen obesity drug developers Novo Nordisk and Eli Lilly.

Carmot had been a rising star in biotech, attracting significant funding, including a $150 million Series E financing round in May 2023. The startup, having raised almost $385 million in total funding, had even flirted with an initial public offering (IPO) under the ticker CRMO., a rarity in the current climate.

CT-388 a valuable asset

Carmot’s lead asset is CT-388, a once-weekly injectable dual GLP-1/GIP receptor agonist for obesity, which has shown upbeat weight loss results in a phase 1b study. In the study, participants experienced more than 8% weight loss at 4 weeks. The drug candidate also appeared to be well-tolerated. In the study, The therapy demonstrated substantial weight loss compared to other…

The future of MDD treatment: A comparative table highlighting the emergence of fast-acting therapies

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The treatment landscape for major depressive disorder (MDD) continues to diversify, and in the coming years, psychedelic options may be available, including COMPASS Pathways COMP360 and the deuterated psilocybin analog CYB003 from Cybin. CYB003 demonstrated a significant -14.08 point reduction in MADRS score, a 53.3% response rate, and a 20% remission rate at a 12mg dose in interim results from its phase 2 study.

The potential of faster-acting more effective therapies

While it is difficult to compare the results from disparate clinical trials, some broad patterns emerge when viewing data from current treatment options and drugs now in clinical development for MDD (see table below). In contrast, newer treatment options, especially psychedelic therapies such as CYB003 and COMP360, demonstrate potentially faster onset and greater magnitude of symptom reduction.

The promise of NMDA and dopamine rec…

Five pharmaceuticals featured on Time’s list of top inventions alongside other medical breakthroughs

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Time magazine’s most recent roundup of 200 inventions included an array of product types, spanning household gadgets and AI applications. Five distinct pharmaceuticals also made the list, not counting a shipping container for biologics and a vaccine for bees. Among the notable pharmaceutical advances on the list are a novel postpartum depression drug, a novel Alzheimer’s therapy, the first FDA-approved RSV vaccine, a blockbuster drug for diabetes and obesity that has driven record-breaking sales for Novo Nordisk. Outside of pharmaceuticals, other medical products that made Time’s list include advanced prosthetic limbs and portable diagnostic devices. Read on to see which pharmaceutical products won the attention of one of the most influential magazines in the U.S.

Zurzuvae, a new PPD treatment option

Zuranolone molecule image from PubChem

Development challenges and regulatory changes for cell and gene therapies

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Cell and gene therapies are the fastest growing area for drug development. The groundwork for this category was laid with the mapping of the human genome in 2003, and the field has developed rapidly in the intervening decades. These powerful therapies have significant potential for the treatment of cancer and other previously “undruggable” diseases. But cell and gene therapies also have unique characteristics that can lead to manufacturing challenges and extended regulatory timelines. As of February 2020, cell and gene therapies account for 12% of the preclinical pipeline and 16% of the clinical pipeline.

Given the scale of investments made to support these development efforts and advancements possible, drug sponsors have looked to the U.S. FDA to consider accelerating gene and cell therapy development. The regulatory body has responded by shifting its departmental structure to st…

Three strikes in pharma: Exploring recent drug withdrawals and clinical trial challenges

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Pharmaceutical companies face a long list of regulatory challenges ranging from patent expiry to bioequivalence and international harmonization. It’s not uncommon for drug makers to withdraw or discontinue drugs after failing to meet clinical requirements or endpoints, resulting in drug withdrawals. On average, life science companies pull roughly 4,500 drugs and devices from the market, many of which are widely used. In recent years, drug companies, for instance, have pulled a growing number of products for nitrosamine contamination. While the FDA can identify safety concerns, it often falls upon the manufacturer to initiate and execute a recall.

Here, we round up three recent examples of recent drug withdrawals where pharma companies either pulled drugs from the market or gave up on developing a drug for a given indication.

Covis Pharma withdraws Makena

The Zug, Switzerland–base…