The healthcare sector has been evolving at a pace like never before, especially in 2023. Post the COVID-19 pandemic, various diseases and infections pose a new challenge for scientists and pharmaceutical companies to develop drugs that are effective and formally backed by legal authorities such as the FDA. FDA approvals play a pivotal role in setting trust among the users and the healthcare providers prescribing medications and therapies to patients to enhance their health and overall patient experience.
2023 has experienced an astonishing 50% upsurge in FDA approvals compared to the previous year. This substantial increase reflects the rapid pace of innovation coupled with the profound impact these approvals have on patients, healthcare providers, and pharmaceutical companies.
The recent drug approvals focused on innovative treatments, novel therapies, and the evolving landscape of the medical field. Some of the most significant FDA approvals of 2023 are as follows:
Topping the list is the approval for Aphexda (motixafortide), a stem cell mobilization agent for patients with multiple myeloma scheduled for autologous stem cell transplantation (ASCT). The Israel-based BioLineRx was approved by the FDA on September 11, 2023, coupled with filgrastim (G-CSF), a granulocyte colony-stimulating factor that stimulates the growth of neutrophils.
Aphexda is BioLineRx’s first FDA-approved medication. The decision made by the FDA was based on the Phase III GENESIS trial where the efficacy of Aphexda was observed in combination with filgrastim and placebo (a substance with no medicinal effects). It was concluded that Aphexda exhibited significant efficacy in mobilizing stem cells for transplantation when tested in combination with filgrastim.
In September, the FDA served a tentative approval to a Pennsylvania–based company Viatris/ abacavir 60 mg/dolutegravir 5 mg/lamivudine 30 mg tablets to cure pediatric HIV patients. President Joe Biden’s Emergency Plan for AIDS Relief (PEPFAR) program has played a significant role in this approval.
According to the press release of Viatris, the formulation met all the quality, safety, and efficacy standards by the FDA. The Viatris has also signed a licensing agreement for pediatric dolutegravir with the Medicines Patent Pool and a development agreement with ViiV Healthcare and the Clinton Health Access Initiative to manufacture and distribute the fixed-dose combination of abacavir/ dolutegravir/lamivudine. It was stated in the same press release that Pediatric HIV patients account for 4% of people living with HIV, they made up 13% of deaths associated with AIDS in 2022 which took place due to a lack of antiretroviral therapy.
Abrysvo by Pfizer, a vaccine used in the treatment of respiratory syncytial virus, received FDA approval to be used in pregnant women to assist prevention of lower respiratory tract disease associated with RSV in infants. The approval was based on the data-driven from the Phase III MATISSE trial. Abrysvo is an unadjuvanted bivalent RSV prefusion F vaccine composed of two proteins that were selected specifically to optimize against the RSV A and B strains. The Abrysvo vaccine shot can be administered to pregnant women at 32 through 36 weeks of gestation to evoke immunity in infants.
At the study’s interim analysis, vaccine efficacy of 81.8% was exhibited by Abrysvo to prevent severe RSV-associated lower respiratory tract disease (LRTD) 90 days after birth. Although a slight decline was exhibited later reducing to 69.4% at 180 days.
Regeneron Pharmaceuticals’ Veopoz secured FDA approval as the first and only treatment specifically to treat CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. This is an ultra-rare hereditary disease that consists of life-threatening gastrointestinal and cardiovascular symptoms.
Veopoz, a complete human monoclonal antibody, is approved for the treatment of adult and pediatric CHAPLE patients aged 1 year and older. Regeneron’s investigational antibody underwent a priority review by the FDA in February 2023. This antibody works by getting attached to the C5 complement factor, thereby disrupting the complement cascade and preventing associated diseases. Regeneron displayed the data retrieved from Phase II/III in order to apply for regulatory approval. The data exhibited rapid and sustained normalization of albumin in all 10 patients at 24 weeks. The treatment also helped patients with improved bowel movements and abdominal pain.
The FDA has approved Regenerons’ cutting-edge eye therapy Eylea (aflibercept), allowing the administration of a higher 8-mg dose. Eylea injections have now been suggested to be administered every four weeks for the first three months across all indications, followed by the new high-dose regimen. Previous approval for Eylea was for a 2 mg dose.
In diabetic retinopathy, the administration can be done every 8 to 12 weeks thereafter, while the dosing interval can stretch up to 16 weeks in patients with wet age-related macular degeneration and diabetic macular edema.
The US Food and Drug Administration (FDA) has granted accelerated approval to Pfizer's Elrexfio, a BCMAxCD3 bispecific antibody, for patients with relapsed or refractory multiple myeloma (RRMM) who have undergone four prior lines of therapy. Pfizer claims that Elrexfio is the first ready-to-use, off-the-shelf, fixed-dose subcutaneous therapeutic that targets the B-cell maturation antigen (BCMA) protein. The approval was based on data from the MagnetisMM-3 study, which showed an overall response rate of 58% in heavily treated RRMM patients who had not received BCMA-directed therapy, with 82% of responders maintaining improvements for at least nine months. In patients with prior exposure to BCMA-directed therapies, Elrexfio elicited an overall response rate of 33%. However, the therapy comes with a warning for cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.
In July, GSK's Jemperli (dostarlimab-gxly) was approved by the FDA as a primary frontline treatment for patients with advanced or recurrent endometrial cancer. This is the first new frontline treatment to be approved in decades for patients with mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H) cancer, according to GSK. The FDA accepted the supplemental BLA to move the drug into the frontline setting nearly two months ahead of schedule on June 6th and approved it on July 31st.
Jemperli is also the first immuno-oncology treatment and PD-1 inhibitor to be authorized for frontline use in this patient population. Jemperli, an anti-PD-1 antibody, was initially approved in April 2021 for recurrent or advanced dMMR endometrial cancer.
In July, Octapharma was granted approval for Balfaxar (prothrombin complex concentrate). Balfaxar is used to reverse the effects of the blood-thinner Warfarin in emergency surgery and invasive procedures, increasing the risk of excess bleeding during surgery. Although Balfaxar met the primary endpoint of hemostatic efficacy in a Phase III study and was non-inferior to the comparator, Kcentra, fatal and non-fatal arterial and venous thromboembolic complications were observed in clinical trials and post-marketing studies. Balfaxar is already marketed as octaplex in Canada and Europe with third approval.
Sanofi and AstraZeneca’s Beyfortus (nirsevimab) achieved FDA approval for the treatment and infants and children who are at severe risk of RSV. Beyfortus, a monoclonal antibody, is the second RSV drug that has been authorized in the United States for children at higher risk.
Synagis (palivizumab) is the first treatment, approved back in 1998. The approval was backed by three late-stage trials that demonstrated the safety and efficacy of the monoclonal antibody. Sanofi mentioned in a press release that the Beyfortus treatment will be available in the U.S. ahead of the 2023/2024 RSV season for children entering their first RSV season and those at severe risk up to 24 months.
The first cellular therapy for type 1 diabetes was approved by the FDA on June 28, 2023. Lantidra (donislecel) which has been developed by Chicago-based CellTrans, is a cell therapy produced from the pancreatic islet cells of deceased donors. It is intended for adult patients who have trouble maintaining appropriate glucose levels due to repeated hypoglycemic episodes.
Pfizer and OPKO Health’s collaboration to develop a human growth hormone brought home an FDA approval which would be known as Ngenla. The treatment has been approved to be used in children whose production of growth hormones is impaired. Pfizer purchased exclusive global commercialization rights for the then-experimental treatment in December 2014 for $295 million upfront and a promise of up to $275 million in milestones.
The FDA gave a nod to Pfizer’s Litfulio(ritlecitinib) to severe alopecia areata treatment, an autoimmune disease, in patients aged 12 years and younger. Litfulo consists of Janus kinase 3 and the tyrosine kinase which is supposed to block the signalling of cytokines and cytolytic activity of T cells implicated in alopecia areata.
FDA approved Sareptas’ Elevidys formerly SRP-9001 for children 4 to 5 years with Duchenne muscular dystrophy (DMD). This was one of the most awaited regulatory decisions of the year. Elevidys is the first-ever gene therapy for DMD, a neuromuscular disease that weakens human muscles with time and atrophy which occurs mostly in young boys. The gene therapy was approved via the FDA’s accelerated approval pathway based on data that exhibited that the developed gene therapy substantially increased the expression of the Elevidys micro-dystrophin protein.
Argenx’s Vyvgart (efgartigimod) is the first subcutaneous injectable for generalized myasthenia gravis (gMG), first approved in 2021 as a one-hour intravenous infusion. Myasthenia gravis (gMG) is a rare neuromuscular disorder. Vyvgart is an antibody fragment that targets neonatal Fc receptors to prevent recycling of immunoglobulin G back into the blood, further reducing the abnormal AChR antibodies present in approximately 85% of gMG patients. By normalizing the levels of these antibodies, the muscular function of gMG patients could be reduced, alleviating symptoms of muscle weakness and fatigue associated with the condition.
These are only some of the key approvals which took place in the second and third quarter of the year, starting from June to October. There are numerous other drugs which received approval by the FDA this year, marking significant developments in the healthcare sector. The healthcare sector is developing rapidly to be prepared to be able tackle every health-related emergency and these drug approvals play a pivotal role in speeding up that development.
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