From Madrigal Pharmaceuticals’ first ever MASH drug Rezdiffra to Eli Lilly’s Alzheimer’s treatment Kisunla, 2024 has already hosted some big drug approvals. But the year isn’t over, and some of the new drugs heading to market promise significant milestones for prominent diseases.
Numerous oncology drugs have been approved in the first half of the year, including two first-in-class therapies in the second quarter, according to the American Association for Cancer Research.
In terms of novel drugs, FDA approvals spiked in 2023 to 55 new treatments (up from 37 in 2022). So far this year, the agency has OK’d 29.
Still, several drug candidates are scheduled for review by the FDA for potential approval in the coming months, including novel drugs and indication expansions that could widen the market and offer breakthroughs in different disease areas.
Drug candidate: KarXT
Owned by: Bristol Myers Squibb
Indication: Schizophrenia
PDUFA date: Sept. 26
Why it’s noteworthy: KarXT is one of the most anticipated potentialapprovals of the year that could be a breakthrough for the schizophrenia space, which has largely lacked innovations for the last 20 years. The antipsychotic offers a new way to treat the disease, which impacts 2.8 million people in the U.S. Other schizophrenia treatments block dopamine receptors while KarXT is the first in a class of muscarinic antagonists, potentially bypassing side effects that often lead to patients stopping treatment .
KarXT came to BMS through a $14 billion acquisition of Karuna Therapeutics at the end of last year, and the drug could pull in $10 billion in peak sales. Executives noted they expect approval of KarXT in the company's most recent earnings call with analysts, and are preparing for launch.
Drug: Dupixent
Owned by: Regeneron Pharmaceuticals and Sanofi
Indication: COPD
PDUFA date: Sept. 27
Why it’s noteworthy: Dupixent, an already approved drug from Regeneron and Sanofi for dermatitis and asthma, may soon be granted an expansion to treat chronic obstructive pulmonary disease, a common lung disease that impacts nearly 12 million Americans. Originally slated for June 27, the expansion faced a delay when the FDA “requested additional analyses on the efficacy of Dupixent” in pivotal trials, Regeneron said in May.
By expanding to COPD, the pharma partners could see a huge revenue boost. Sanofi has pegged the sales numbers above $14 billion at its peak with the COPD expansion, Reuters reported last year. Dupixent gained European approval for COPD in July, making it the “first-ever targeted therapy for patients” with the disease, Sanofi said. Sanofi and Reneron might not be alone in the COPD market for long, however, with AstraZeneca and Amgen following closely behind with a clinical-stage biologic of their own.
Drug candidate: Acoramidis
Owned by: BridgeBio Pharma
Indication: ATTR cardiomyopathy
PDUFA date: Nov. 29
Why it’s noteworthy: The day after Thanksgiving could offer BridgeBio a lot to be thankful for as its lead candidate acoramidis faces a potential FDA green light. The candidate is an oral drug for patients with ATTR cardiomyopathy, a condition that leads to heart failure. The disease is often underdiagnosed due to its subtle symptoms, but it has become an area of interest for drug research. The only other treatments on the market: Pfizer’s Vyndamax and Vyndaqel — together brought in nearly $2.5 billion during the first six months of 2024.
BridgeBio secured $310 million in up front and near-term milestone payments from Bayer earlier this year as part of a licensing deal for the German pharma giant to market the drug in Europe. Some estimates suggest acoramidis could rake in up to $2 billion in peak sales, and BridgeBio is in line for more milestone payments along the way.
