The US Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) approved 50 novel drugs across 2024.
According to its report Advancing Health Through Innovation: New Drug Therapy Approvals CDER detailed the 50 drugs that were never before approved or marketed in the US. CDER added: “We also made other important approval decisions, such as approving previously approved drugs for new uses and broader patient populations.”
Approvals also included therapies for several diseases and conditions with few to no treatment options.
Therapies with orphan drug designations accounted for over 50% of CDER’s novel drug approvals. In addition, CDER highlighted that a record total of 18 biosimilars for eight reference products were approved during 2024, more than any previous year. CDER also said that 24 of the 50 novel drugs approved in 2024 are identified as first-in-class. In addition, 34 of the 50 novel drugs approved in 2024 were approved in the US before any other country.
The rare diseases receiving drug approval included:
• Neimann-Pick disease type C, a genetic disease that results in progressive neurological symptoms and organ dysfunction.
• Duchenne muscular dystrophy, a severe, inherited disease that causes progressive muscle weakness and muscle wasting.
• Primary biliary cholangitis, a chronic autoimmune disease that damages the bile ducts in the liver.
• Familial chylomicronemia syndrome, a life-threatening disease that prevents the body from digesting fats.
• Classic congenital adrenal hyperplasia, a group of genetic disorders that affect the adrenal glands and cause a deficiency in cortisol and/or aldosterone.
There were also approvals for therapies for rare cancers or tumors, including:
• HER2-positive (IHC3+) tumors, which have higher levels of the human epidermal growth factor receptor 2 (HER2) protein that enables faster growth and spread.
• Grade 2 astrocytoma, a slow-growing brain tumor that can become more aggressive over time.
• Gastroesophageal junction adenocarcinoma, a rare type of cancer that starts in the gastroesophageal junction.
• Small cell lung cancer.
Reducing the time to get new drugs to the market has been a focus of CDER during 2024 and the report highlighted several strategies employed to achieve this. These strategies included priority review, with 28 of the 50 novel drugs approved designated priority review. A drug receives a priority review if CDER determines that the drug treats a serious condition and, if approved, would provide a significant improvement in safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions. A priority review application is one on which CDER sets a goal to take action within six months of filing (compared to a target date of 10 months under standard review).
A further seven drugs were approved via the accelerated approval pathway. This route brings certain drugs for unmet medical needs to market on a faster timeline than would be possible following the traditional approval pathway. Accelerated approval may be an option for a new drug intended to treat a serious condition that offers a meaningful advantage over available therapies, CDER added.
New formulations also made up a significant number the 2024 approvals with CDER noting: “New formulations of approved drugs can offer significant therapeutic advances. Similarly, new dosage forms (such as from a capsule to a chewable tablet for those unable to swallow pills) can help increase adherence, make sure patients take the proper dose, and improve quality of life for patients who must use the medication on a prolonged basis. Making a drug available as an over-the-counter product can also increase patient access to therapies.”
Big players including Pfizer, Merck and Amgen received approvals for drugs treating heamophilia, pulmonary aterial hypertension, and advanced small-cell lung cancer respectively. Eli Lilly’s drug for early Alzheimer’s disease also received approval. With limited options for treating Alzheimer’s this development has been widely welcomed in the US.
Smaller companies including Allecra Therapeutics; treating complicated urinary tract infections including the kidney infection pyelonephritis; Iovance Biotherapeutics, for the first cell therapy to treat advanced melanoma; and BridgeBio Pharma’s treatment for patients with the rare heart disease transthyretin amyloid cardiomyopathy also received product approvals.
Similar developments were seen in the European Union with 114 new medicines being approved for human use by the European Medicine Agency (EMA). This number included 48 drugs containing a completely new active substance with 16 for the treatment of rare diseases. The EMA noted that it is the first time since 2009 that it has been able to deliver more than 100 positive opinions.
There were also approvals for veterinary medicines, with 23 recommendations for new medicines for animals, of which 12 are vaccines. Highlighting several firsts, the EMA added that it issued a positive opinion for marketing authorisation in the European Union for the first medicine shown to slow the progression of Alzheimer's disease in certain patients, as well as for the first vaccine to protect adults from Chikungunya, a virus transmitted to people by mosquitoes.
With AMR a continued threat, the EMA’s recommendation for marketing authorisation for a new antibiotic to fight infections caused by multidrug-resistant bacteria was also notable.
In order of approval, the 50 novel drugs approved by the FDA by brand name with active ingredient in brackets are:
Zelsuvmi (berdazimer)
Exblifep (cefepime, enmetazobactam)
Letybo (letibotulinumtoxinA-wlbg)
Tevimbra (tislelizumab-jsgr)
Rezdiffra (resmetirom)
Tryvio (aprocitentan)
Duvyzat (givinostat)
Winrevair (sotatercept-csrk)
Vafseo (vadadustat)
Voydeya (danicopan)
Zevtera (ceftobiprole medocaril sodium)
Lumisight (pegulicianine)
Anktiva (nogapendekin alfa inbakicept-pmln)
Ojemda (tovorafenib)
Xolremdi (mavorixafor)
Imdelltra (tarlatamab-dlle)
Rytelo (imetelstat)
Iqirvo (elafibranor
Sofdra (sofpironium)
Piasky (crovalimab-akkz)
Ohtuvayre(ensifentrine)
Kisunla (donanemab-azbt)
Leqselvi(deuruxolitinib)
Voranigo(vorasidenib)
Yorvipath(palopegteriparatide)
Nemluvio(nemolizumab-ilto)
Livdelzi(seladelpar)
Niktimvo(axatilimab-csfr)
Lazcluze(lazertinib)
Ebglyss (lebrikizumab-lbkz)
Miplyffa(arimoclomol)
Aqneursa (levacetylleucine)
Cobenfy (xanomeline and trospium chloride)
Flyrcado(flurpiridaz F 18)
Itovebi (inavolisib)
Hympavzi(marstacimab-hncq)
Vyloy (zolbetuximab-clzb)
Orlynvah(sulopenem etzadroxil, probenecid)
Revuforj(revumenib)
Ziihera (zanidatamab-hrii)
Attruby (acoramidis)
Rapiblyk(landiolol)
Iomervu (iomeprol)
Bizengri(zenocutuzumab-zbco)
Unloxcyt(cosibelimab-ipdl)
Crenessity(crinecerfont)
Ensacove(ensartinib)
Tryngolza(olezarsen)
Alyftrek (vanzacaftor, tezacaftor, and deutivacaftor)
Alhemo (concizumab-mtci)
Further reading:
• AstraZeneca at 25: ‘The opportunity and the potential is enormous’
• Pharma R&D: Who is really setting the pace?
• Small molecule drugs for previously untreatable diseases
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