Every year, the U.S. Food and Drug Administration (FDA) approves a wide range of new drugs and biological products. This year is no exception, with some long-awaited blockbuster approvals and a few unexpected additions. 

In 2023 to date, 51 novel drugs have been approved by the FDA to manage a broad spectrum of medical conditions – from Alzheimer’s disease and cancer to migraines, menopausal symptoms and post-partum depression. We highlight 10 from the list that are set to have a significant impact. 

We begin our list with three breakthrough drugs that are set to transform women’s healthcare. 

ORSERDU (elacestrant) 

to manage advanced metastatic breast cancer 

Around 10 million people worldwide will die from cancer every year and approximately 685,000 of those deaths will be due to breast cancer.  

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Orerdu (elacestrant) is a selective oestrogen receptor degrader (SERD) marketed by Stemline Therapeutics – a subsidiary of Menarini in the U.S. It is the first SERD to be approved for the management of advanced or metastatic breast cancer in postmenopausal women or adult men. The treatment is specifically intended for individuals with oestrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer that has progressed after at least one round of endocrine therapy and is characterised by ESR1 mutations.  

Positive feedback from clinicians has resulted in a strong initial uptake of Orserdu, with  2023 sales projected to reach at least $175m.  

ZURZUVAE (zuranolone) – to treat postpartum depression 

Postpartum depression (PPD) may occur during pregnancy or in the first year after giving birth and affects up to one in seven women.  

Zurzuvae (zuranolone) is a gamma-aminobutyric acid (GABA) positive allosteric modulator developed by Sage Therapeutics and Biogen. The FDA has approved it as the first oral treatment for postpartum depression in women. It is taken daily for a duration of 14 days and its mood-enhancing effects are felt within just three days.  

“The only alternative drug treatment currently sanctioned for this condition,” observes Surbhi Gupta, senior industry analyst, growth opportunity analytics – HLS, at Frost & Sullivan, “involves the administration of an intravenous medication, which necessitates a hospital-like stay over 60 hours. A rapidly acting oral medication could potentially mitigate the subsequent consequences of postpartum, particularly in relation to the maternal-infant bond.” 

According to GlobalData analysts, Zurzuvae will be particularly attractive for patients who may have concerns about longer term medication entering their breast milk. 

GlobalData is the parent company of Pharmaceutical Technology.

However, although the approval for PPD is significant, the FDA rejected ZURZUVAE for the treatment of major depressive disorder (MDD), which would have opened up a huge potential market. The drug is a central nervous system depressant and has the potential for abuse, so it will have to undergo a scheduling review before it can enter the market.  

Sales projections for Zurzuvae vary, with some analysts suggesting total revenues of between $250m to $500m for PPD sales and $1bn plus for the future MDD market. 

VEOZAH (fezolinetant) – to manage menopausal hot flushes 

Vasomotor symptoms (hot flushes) affect around 80% of menopausal women and can include debilitating periods of sweating, flushing and chills. 

Vezoah (fezolinetant) is a neurokinin (NK) 3 antagonist marketed by Astellas Pharma. The drug is the first oral nonhormonal neurokinin 3 (NK3) receptor antagonist authorised for the management of menopause-induced moderate to severe hot flushes. It works by binding to and blocking the activities of the NK3 receptor, which plays a role in the brain’s regulation of body temperature.  

VEOZAH includes a warning for potential liver injury and routine bloodwork should be performed every three months for the first nine months of using the medication.  

Estimated global sales: $2.5bn by 2029, as per GlobalData 

ZAVZPRET (zavegepant) – to treat migraine 

Migraine is a debilitating neurologic disorder that affects more than one billion people globally every year; it has a particularly high prevalence among young adults and women. 

Zavzpret (zavegepant) is a calcitonin gene-related peptide (CGRP) antagonist nasal spray developed by Pfizer, in collaboration with Bristol Myers Squibb. It is the first and only non-oral formulation of a CGRP antagonist and is proven to prevent and treat both chronic and episodic migraine, with or without aura, in adults. Approved by the FDA in March, Zavzpret provides an alternative treatment option for people who need pain relief and cannot take oral medications due to migraine-related nausea or vomiting.  

Key opinion leaders previously interviewed by GlobalData believed that the intranasal formulation would give Zavzpret a significant edge over competitors in the same drug class, namely AbbVie’s Ubrelvy (ubrogepant) and Biohaven’s Nurtec (rimegepant). 

Estimated global sales: $725m million for 2029, as per GlobalData 

VOWST (fecal microbiota spores) – to prevent recurrent Clostridioides difficile infection 

C. diff(also known as C. difficile or CDI) is a bacterium that causes diarrhoea and colitis. It is responsible for almost half a million infections in the U.S. each year, can recur within weeks, and one in 11 people over the age of 65 diagnosed with a healthcare-associated C. diffinfection die within one month. 

Vowst (fecal microbiota spores) is a microbiome modulator developed by US-based biotech company Seres Therapeutics and marketed by Nestlé. Vowst or SER-109, is a microbiome therapeutic used to prevent the recurrence of CDI in individuals aged 18 years and older who have undergone antibacterial treatment for recurrent CDI.  

Although antibiotics are effective against CDI, they also disturb the gut microbiome’s sensitive ecosystem. Vowst, the first FDA-approved oral microbiome therapy, utilises purified firmicutes spores, a beneficial microbiome, to competitively shift C. diff and bring the gut microbiome back into healthy balance. A clinical study demonstrated that six months after taking VOWST, most people had not had a recurrence of CDI. 

Estimated global sales: $442 million for 2029, as perGlobalData 

LEQEMBI (lecanemab)– to treat Alzheimer’s disease 

Globally, the incidence of Alzheimer’s and other dementias is on the increase. More than 55 million people worldwide are living with dementia and this figure is projected to rise to 139 million in 2050. 

Leqembi (lecanemab) is an anti-beta-amyloid (Abeta) protofibrils mAb marketed by Eisai and administered via intravenous infusion. The drug targets and removes beta-amyloid from the brain, a defining feature of the disease, thus reducing cognitive and functional decline in people living with early Alzheimer’s.  

 “It is the second of a new category of medications approved for Alzheimer’s disease that target the fundamental pathophysiology of the disease,” says Gupta. 

Some physicians have commented that the drug’s effect on the disease may not be clinically effective enough to warrant the risk of brain swelling, which is one of the potential side effects. However, with trial data showing Leqembi could slow cognitive decline by 27%, many patients with early Alzheimer’s are keen to access it. Leqembi was approved by the FDA in January, but is still awaiting approval by the European Medicines Agency.  

Despite that, GlobalData analysts predict this breakthrough drug will be a blockbuster.  

Estimated global sales: $12.9bnby 2028, as per GlobalData 

Leqembi faces competition from Eli Lilly’s donanemab, a similar monoclonal antibody targeting amyloid beta plaques, for the treatment of early Alzheimer’s disease. The approval for this drug is expected in early 2024. 

SKYCLARYS (omaveloxolone) – to treat Friedreich’s ataxia 

Friedreich’s ataxia is a rare hereditary neurodegenerative disorder usually identified in adolescence. It causes a decline in coordination, muscle strength, and energy levels that typically leads to premature death.  

Skyclarys (omaveloxolone) is a nuclear factor erythroid derived 2 (Nrf2) activator developed by Reata Pharmaceuticals. The FDA granted approval for SKYCLARYS in February to treat Friedreich ataxia in those over 16 years of age. The drug is the first and only approved therapy for this condition and has been granted fast track and rare paediatric disease designation by the FDA.  

Skyclarys, initially developed through a partnership between Reata Pharmaceuticals and AbbVie, was later repurchased by Reata in 2019. Biogen acquired Reata in 2023 and is currently engaged in the marketing of Skyclarys. Reata has applied for approval in Europe and intends to launch there in early 2024. 

Estimated global sales: $1.3bnby 2029, as perGlobalData 

ELEVIDYS (delandistrogene moxeparvovec) – to treat Duchenne muscular dystrophy 

Duchenne muscular dystrophy (DMD) is a genetic disorder that causes progressive muscle degeneration due to alterations of dystrophin, a protein that keeps muscle cells intact. Prevalence is approximately six per 100,000, with early childhood onset. 

Elevidys (delandistrogene moxeparvovec) is a micro-dystrophin gene therapy marketed by Sarepta Therapeutics. It is the only gene therapy that has been authorised for the treatment of DMD and was granted accelerated approval by the FDA having shown that it increased the marker micro-dystrophin in patients treated.  

Elevidys is used to treat ambulatory children aged four to five years old with DMD who have a confirmed mutation in the dystrophin gene. Further approval for the drug may depend on the verification and description of clinical benefit in additional confirmatory trials.  

“Existing treatment methods primarily focus on managing the symptoms of the disease rather than targeting its fundamental genetic basis,” says Gupta. “Several other manufacturers, including Pfizer, are developing gene therapies for the treatment of DMD such as fordadistrogene movaparvovec.” 

Estimated global sales: $5.6bn by 2029, as perGlobalData 

POMBILITI (cipaglucosidase alfa) – to treat Pompe disease 

Pompe disease (acid alpha-glucosidase [GAA] deficiency) is an inherited disorder characterised by breathing difficulties and muscle weakness. Incidence is approximately 1 in 40,0000. 

Pombiliti (cipaglucosidase alfa) is used to treat adults with late-onset Pompe disease (LOPD). It is an alpha glucosidase long-term enzyme replacement therapy and is used with the enzyme stabiliser Opfolda (miglustat) as a unique two-component therapy. In clinical trials, patients treated with Pombiliti plus Opfold walked on average 21 metres further from baseline than those not given the drug. Pombiliti was approved by the FDA in September and will be distributed and marketed by Amicus Therapeutics.  

Pombiliti (in combination with Opfolda) has also been approved for the treatment of adults with LOPD in the European Union and the United Kingdom.  

Estimated global sales: $453m by 2029, as perGlobalData 

NARCAN NASAL SPRAY (naloxone hydrochloride) – to treat opioid overdose 

While opioids can be helpful in the management of chronic pain, their addictive nature and illicit use has fuelled the U.S. opioid crisis. Drug overdose continues to be a major public health issue in the U.S.; a reported 111,355 fatal overdoses occurred in the 12-month period ending in April 2023, most involving illicit and dangerous versions of synthetic opioid, fentanyl. 

Narcan is an opioid antagonist and works by blocking the effects of opiates to alleviate dangerous symptoms caused by high levels of opiates in the bloodstream. It is the standard treatment for overdose from drugs such as heroin, oxycodone, and fentanyl. 

The FDA previously approved NARCAN nasal spray in 2015 for prescription use, but crucially this year’s approval allows over-the-counter (OTC) use – the first naloxone product approved for use without a prescription. Given the scope of the opioid crisis, this decision will have a far-reaching impact, increasing access to the public through local stores, petrol stations and other outlets and potentially saving more lives.