The year 2024 was marked by some landmark drug approvals in the areas of schizophrenia, metabolic dysfunction-associated steatohepatitis (MASH), chronic obstructive pulmonary disease (COPD) and Alzheimer’s disease. The incoming Trump administration, monkeypox outbreak and drug price negotiations in the US also created big news. Here is PharmaCompass’ compilation of the top 10 news from Phispers of 2024.
I. BMS’ US$ 14 bn Karuna bet pays off with landmark FDA approval for schizophrenia med Cobenfy
The US Food and Drug Administration’s groundbreaking approval of Bristol Myers Squibb’s Cobenfy to treat schizophrenia has
made it to our number one spot. BMS had acquired
Cobenfy through its US$ 14 billion purchase of Karuna Therapeutics, announced in December 2023.
Cobenfy is the first-ever antipsychotic that targets cholinergic receptors. Cobenfy’s new approach has experts excited. Its peak sales are expected
to come in at US$ 7.5 billion a year.
While BMS’ Karuna buyout paid off, AbbVie’s US$ 8.7 billion acquisition of Cerevel Therapeutics was dampened when their lead candidate and schizophrenia drug emraclidine failed to meet phase 2 endpoints in two pivotal trials.
II. 2024’s biggest deal, US$ 16.5 bn Catalent buyout by Novo’s parent, cleared after intense scrutiny
On number two spot is Novo Nordisk Foundation’s acquistion of Catalent via its investment arm Novo Holdings for US$ 16.5 billion, announced in February 2024. Novo Holdings plans to sell three of Catalent’s “fill-finish” sites to Novo Nordisk for US$ 11 billion.
Easily the biggest biopharma acquisition of last year, the deal faced criticism from lawmakers and consumer groups for its potential anti-competitive effects, particularly in the glucagon-like peptide-1 (GLP-1) drug market. Its main competitor Eli Lilly called for scrutiny by competition regulators. However, in December, both the EU regulator and the US Federal Trade Commission gave it the greenlight.
III. Madrigal’s Rezdiffra becomes first FDA-approved drug for liver disease MASH
For quite some time, the drug development field for the liver
condition MASH had been a graveyard for failed programs. In 2024, the field
celebrated a hurrah moment when Madrigal’s oral drug Rezdiffra became the first treatment in the US for
adults with this common fatty liver disease. The approval has opened a multi-billion dollar opportunity for Madrigal with the American Liver Foundation CEO Lorraine Stiehl calling it a “game-changing” moment.
IV. COPD patients get two new treatments — Verona’s Ohtuvayre and Sanofi-Regeneron’s Dupixent
The COPD landscape saw remarkable advances in 2024. FDA
approved Verona Pharma’s Ohtuvayre, which brings the first new mechanism of action in
over two decades for the treatment
of COPD. This dual-action, inhaled medication serves as both a bronchodilator
and an anti-inflammatory agent, offering a comprehensive approach to symptom
management. Adding to this progress, Sanofi and Regeneron’s Dupixent achieved a historic milestone by becoming the first biologic approved for treating
COPD. Over 390 million people are living with COPD worldwide.
V. Novartis inks 20 deals in
2024, promises over US$ 25 billion in biobucks, lays bets on radiopharmas
Novartis has been investing in early-stage science, buying out companies in the sub-US$ 5 billion range. The Swiss drugmaker Novartis inked over 20 deals in 2024, paying over US$ 5.5 billion upfront and promising over US$ 25 billion in biobucks.
The company has particularly focused on radiopharmaceuticals and
molecular glue degraders. It acquired Mariana Oncology to strengthen its
radioligand therapy (RLT) pipeline, and also inked RLT deals with PeptiDream and Ratio Therapeutics.
VI. Despite political
pressure, FDA rejects first MDMA-assisted therapy to treat PTSD; Lykos faces
expanded probe
In August, FDA declined to approve an MDMA-assisted therapy from Lykos Therapeutics to treat post-traumatic
stress disorder (PTSD). In the lead-up to the FDA decision, 80 members of
Congress from both sides of the aisle had urged US President Joe Biden and
the FDA to consider further studies on the therapy. Not satisfied with just declining Lykos’ MDMA-assisted therapy for PTSD, FDA had expanded its investigation into the clinical
trials that tested it. Lykos CEO Amy Emerson stepped down after reducing its workforce
by 75 percent. Additionally, its founder
Rick Doblin exited its board.
VII. Alzheimer’s gets two new treatments as FDA approves Lilly’s Kisunla, Alpha Cognition’s Zunveyl
FDA finally approved Eli Lilly’s Alzheimer’s drug Kisunla in July, after delaying action on it in March. This approval, along with that of Biogen and Eisai’s Leqembi in 2023, mark successes in treating the mind-wasting disease after three decades of failed efforts.
FDA also approved Alpha Cognition’s Zunveyl to treat mild-to-moderate Alzheimer’s disease. Zunveyl is a prodrug of Alzheimer’s medication galantamine, and is to be taken orally twice a day. Alpha said Zunveyl is
designed to address the tolerability issues with galantamine.
VIII. IRA price talks
conclude; Stelara, Enbrel, Eliquis to bring 51.4% of Medicare savings
After filing futile lawsuits questioning the
constitutional validity of the Medicare drug price negotiations under the
Inflation Reduction Act (IRA), all drug companies came to the bargaining table.
In August, the Biden-Harris Administration announced it had reached agreements for
lower prices for all 10 drugs selected for negotiations
under the IRA. The new prices come into effect from January 1, 2026. A
Brookings Institution report said that just three
drugs will account for over half of the expected US$ 6 billion savings in
2026. J&J’s Stelara (US$ 1.4 billion), Amgen’s Enbrel (US$ 1.1 billion),
and BMS’ Eliquis (US$ 856 million) are
expected to account for US$ 3.28 billion or 51.4 percent of Medicare savings.
IX. Trump’s picks for new administration send shockwaves across markets, stocks of vaccine makers tumble
Donald Trump’s picks for his new administration not only created big news, but also impacted the stock markets. His selection of Robert F. Kennedy Jr.,
an anti-vaccine activist, to lead the Department of
Health and Human Services (HHS), sent shares of several vaccine
manufacturers plummeting.
In the US, stocks of Pfizer, Moderna, and Novavax dipped. In Europe,
stocks of BioNTech, GSK, and Bavarian Nordic tumbled. Trump has nominated Mehmet Oz, a celebrity
TV host, to run the Centers for Medicare and Medicaid Services (CMS), which
oversees health insurance.
X. New mpox outbreak in Africa sparks global response; FDA approves Emergent’s vaccine to treat mpox
In
August, a new mpox virus clade put Africa in the throes of an mpox outbreak. The
WHO declared it a public health emergency of international concern. Emergent BioSolutions said it will donate 50,000 doses of its smallpox
vaccine ACAM2000. Multiple countries responded with donations of Bavarian Nordic’s Jynneos, the only widely-approved mpox vaccine at the time.
In early September, the FDA approved Emergent’s ACAM2000 as the second mpox vaccine.
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