The first half of 2024 saw a significant slowdown in approvals of new drugs and biologics by the US Food and Drug Administration (FDA) compared to the same period last year.FDA’s Center for Drug Evaluation
and Research (CDER) approved 21 drugs in H1 2024, reflecting a 19 percent decrease from the 26 approvals granted in H1 2023. Of them, 81 percent (17) were first-in-class drugs (therapies that use a new and unique mechanism of action), while small molecules made up for 67 percent (14) of the total drugs approved.Similarly, the Center for Biologics
Evaluation and Research (CBER) granted approvals to only eight biologics, as compared to 10 in H1 2023.Health Canada also saw a drop in drug approvals as only 10 drugs were okayed in H1 2024, as opposed to 13 approvals in H1 2023.The European Medicines Agency (EMA) saw a marginal rise in drug authorizations at 15 for H1 2024 as compared to 14 approvals in H1 2023. Interestingly, the EMA also saw a surge in pending decisions (applications under review) — from two in H1 2023 to 14 in H1 2024.View New Drug Approvals in H1 2024 with Estimated Sales (Free Excel Available)Merck, Madrigal, Verona bag approvals for breakthrough meds; Lilly’s donanemab okayedThe first half saw some closely watched drugs win regulatory
approvals. FDA approved a breakthrough therapy from Merck — Winrevair (sotatercept) — that treats adults with hypertension caused by the constriction
of arteries in the lungs, known as pulmonary arterial hypertension (PAH).Merck had acquired Winrevair
through its US$ 11.5 billion acquisition of Acceleron Pharma in 2021. The therapy is set
to generate nearly US$ 3 billion in global peak sales by 2028. Another breakthrough therapy approved in H1 2024 is Madrigal’s Rezdiffra (resmetirom), the first FDA-approved treatment for adults with the common fatty liver disease — nonalcoholic steatohepatitis (NASH). Rezdiffra is expected to touch sales of US$ 2.1 billion by 2028.The agency also approved the first maintenance treatment for chronic obstructive pulmonary disease (COPD) in over 20 years — Verona’s Ohtuvayre. The drug has a novel
mechanism of action and is the first inhaled maintenance treatment
for COPD. Approved in June by the FDA, Ohtuvayre is forecast to bring in global
sales of US$ 1.5 billion by 2030.The approval of Eli Lilly’s donanemab was surprisingly delayed, and finally came through on
July 2 after an FDA advisory committee voted unanimously in favor of its
benefits outweighing its risks. To be sold as
Kinsula, the
Alzheimer's drug is estimated to bring in US$ 2.2 billion in sales by 2028.Across the pond, EMA approved Novo Nordisk’s weekly insulin injection Awiqli (insulin icodec). The replacement insulin in Awiqli acts in the same way as the body’s own insulin and helps glucose enter cells from the blood. Meanwhile, FDA rejected this once-a-week insulin
earlier this month and has requested information related to the manufacturing
process.View New Drug Approvals in H1 2024 with Estimated Sales (Free Excel Available) ImmunityBio, Geron, Day One
win approvals for their oncology drugsIn what marks the first approval for ImmunityBio, FDA greenlit Anktiva
(nogapendekin alfa inbakicept-pmln) as part of a combination therapy to treat a type of bladder cancer. Anktiva is a next-generation immunotherapy that creates long-term immunity by activating the so-called natural killer (NK) cells and T-cells. It will compete with Merck’s Keytruda. Anktiva’s yearly sales by 2030 are expected to be around
US$ 1.7 billion.BeiGene’s PD-1 blocker Tevimbra (tislelizumab) got the go-ahead from the FDA as the
treatment for adult patients with unresectable or metastatic esophageal
squamous cell carcinoma (ESCC) after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor. Tevimbra’s 2028 global sales are forecast to bring in US$ 1.6 billion.FDA signed off on Geron’s Rytelo (imetelstat) for treating transfusion-dependent
anemia in patients with low- to
intermediate-risk myelodysplastic syndromes (MDS), a group of blood cancers. This was Geron’s maiden approval and Rytelo is expected to bring in US$ 1.3 billion by 2030.Day One Biopharmaceuticals’ Ojemda (tovorafenib) was granted FDA’s accelerated approval to treat certain types of pediatric brain
cancer. This is the first FDA approval of a systemic therapy for treating
what is the most common form of childhood brain tumor, including fusions.
Ojemda is forecast to bring in US$ 1 billion in sales by 2030.FDA granted accelerated approval to Amgen’s Imdelltra (tarlatamab-dlle) for adults in advanced stages of small cell lung
cancer (SCLC) that has proven to be hard to treat or has worsened
despite platinum-based chemotherapy. Imdelltra is expected to bring in
annual sales of US$ 975 million by 2030.View New Drug Approvals in H1 2024 with Estimated Sales (Free Excel Available) Infectious disease drugs from
Basilea, Merck, rare disease med from Ipsen bag
approvalsAfter oncology, infections and infectious diseases, and rare
diseases were the two therapeutic areas that saw the second and third most approvals,
respectively. FDA approved Basilea Pharmaceutica’s Zevtera (ceftobiprole medocaril
sodium for injection), an antibiotic for bacterial infections
including multidrug-resistant strains.The US agency also approved Merck’s next-generation vaccine designed to protect adults from pneumococcus bacteria that causes serious illnesses and pneumonia. The jab, known as Capvaxive, helped produce an immune response against all 21 variations (serotypes) of the bacteria that it targeted. These 21 strains account for about 85 percent of invasive pneumococcal disease cases in adults aged 65 and above. FDA also approved Moderna’s mRESVIA, a messenger RNA-based (mRNA) respiratory syncytial virus (RSV) vaccine, to protect adults aged 60 years and
older from lower respiratory tract disease caused by the syncytial
virus. This is the first non-Covid mRNA vaccine
to be approved in the US.The agency granted accelerated approval to Ipsen’s Iqirvo (elafibranor) to treat primary
biliary cholangitis (PBC), a rare liver disease. This is the first new
medicine approved in nearly a decade for the treatment of PBC. Orchard Therapeutics’ Lenmeldy secured FDA approval to become the first gene therapy in the US for a rare pediatric disorder, known as metachromatic
leukodystrophy (MLD). The debilitating hereditary disease affects the
brain and the nervous system and causes loss of cognitive and motor functions
and early death.View New Drug Approvals in H1 2024 with Estimated Sales (Free Excel Available) Our viewThe increased momentum of drug approvals witnessed after the pandemic appears to have slowed down, but what’s encouraging is the increase in first-in-class therapies, cancer drugs and promising new treatment options for a range of conditions such as PAH, NASH, and COPD.The
second half has already kicked off with the approval of Lilly’s donanemab. And there are several pathbreaking drugs likely to be approved soon, such as Karuna Therapeutics’ schizophrenia drug KarXT and BridgeBio’s heart drug acoramidis. There is every possibility that new drug approvals will spring back up in H2 2024.
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