The United States Food & Drug Administration (FDA) granted approval to a number of groundbreaking drugs during the month of May. These FDA-approved drugs represent significant milestones, as some of the drugs became the first of their kind to receive recognition from the FDA. Below, we’ll discuss the complex journey of FDA-approved drugs and share the top eight drugs to gain FDA approval in May 2023, highlighting their notable breakthroughs and the impact they hold on the future of healthcare.
The Complex Journey of FDA-Approved Drugs: Navigating the Path to Patient Safety
Securing FDA approval for a new drug is a formidable challenge, and the road to success is often long and arduous. The FDA plays a pivotal role in safeguarding public health by meticulously evaluating pharmaceuticals and medical devices before they reach the market. This rigorous process ensures that drugs are safe, effective, and meet stringent quality standards. Multiple factors contribute to the difficulty of obtaining FDA approval. From preclinical research and extensive clinical trials to meticulous documentation and stringent regulatory requirements, drug developers face numerous hurdles. The FDA’s unwavering commitment to patient safety demands robust evidence of a drug’s benefits outweighing its risks. Consequently, the arduous journey of FDA-approved drugs is a testament to the agency’s dedication to protecting public health and providing patients with safe and effective treatments. The number of FDA-approved drugs can vary from year to year. On average, there are around 20-50 new FDA-approved drugs annually. This number can fluctuate depending on various factors, including the complexity of the drugs, the therapeutic area they target, and the availability of innovative treatments in specific years. May 2023 was a successful month for drug development, as 8 new FDA-approved drugs were announced.
8 FDA-Approved Drugs from May 2023
1. Elfabrio Injection
Treatment for: Adult Patients Diagnosed with Fabry’s Disease
Manufactured by: Protalix Biotherapeutics
Date of Approval: May 9th, 2023
Elfabrio is an FDA-approved drug alternative enzyme replacement therapy specifically developed to address the needs of adult patients diagnosed with Fabry’s disease. Fabry’s Disease, also referred to as Anderson-Fabry disease, is an inheritable rare disease characterized by a deficiency in the alpha-galactosidase-A enzyme (α-Gal A), which leads to the buildup of a fatty substance called globotriaosylceramide (Gb3) in the body’s blood vessels and tissues. This buildup can cause various symptoms and potentially result in organ damage or failure. Elfabrio works as a bi-weekly infusion treatment designed to provide the body with a supplemental source of α-Gal A. The treatment uses a chemically modified and stabilized version of α-Gal A derived from plant cells. Once infused, these modified enzyme molecules work to break down the accumulated Gb3 in various tissues and organs. By reducing the burden of Gb3 buildup, Elfabrio has shown to decrease the adverse effects of Fabry disease on the body and potentially slow down the progression of the disease.
2. Veozah fezolinetant
Treatment for: Vasomotor Symptoms due to Menopause
Company: Astellas Pharma US, Inc.
Date of Approval: May 12, 2023
Veozah is a groundbreaking non-hormonal treatment used to reduce the severity of vasomotor symptoms (VMS) that occur during menopause, such as hot flashes and night sweats. VMS is experienced when the balance between estrogen hormones and a brain chemical called neurokinin B (NKB) is disrupted during the menopausal transition. Traditionally, hormonal therapies like estrogen and progesterone have been used to manage VMS. However, these therapies can pose increased risks for individuals with a history of breast cancer or cardiovascular conditions. Veozah offers an alternative solution for managing VMS without the use of hormonal therapies.
As a first-of-its-kind non-hormonal neurokinin 3 (NK3) receptor antagonist, Veozah works by targeting the disruption between estrogen hormones and NKB, effectively restoring balance and reducing the frequency and severity of hot flashes and night sweats. This significant development provides menopausal women with a safe and effective alternative to traditional hormonal treatments, meeting their needs for symptom relief while minimizing potential health risks. Veozah’s approval is supported by results from three Phase III clinical trials, which included 3,000 individuals from across the United States, Canada, and Europe.
3. Vyjuvek
Treatment for: Dystrophic Epidermolysis Bullosa
Company: Krystal Biotech, Inc.
Date of Approval: May 19th, 2023
Vyjuvek, another first-of-its-kind drug approved by the FDA in May, offers new hope for patients with dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder characterized by connective tissue abnormalities caused by mutations in Collagen Type VII Alpha 1 Chain (COL7A1), a protein-coding gene responsible for the assembly of a larger protein called type VII collagen (COL7). COL7 is a main component in the structure of anchoring fibrils, which hold the epidermis and dermis together by connecting the epidermal basement membrane to the dermis.
Individuals with DEB have highly fragile skin that easily blisters with minor friction or injury. With more than 700 known mutations, symptom severity varies widely. Vyjuvek utilizes a modified herpes simplex virus type 1 (HSV-1) vector to deliver functional copies of the COL7A1 gene, enabling the production of COL7. Administered topically during bandage changes, Vyjuvek not only accelerates wound healing but also strengthens the skin layers. Its approval represents a significant milestone in DEB management by addressing the underlying genetic cause and enhancing wound healing. Vyjuvek is expected to become available in the United States in the third quarter of 2023.
4. Epkinly
Treatment for: Diffuse Large B-cell Lymphoma (DLBCL).
Company: AbbVie Inc.
Date of Approval: May 19th,2023
The FDA’s approval of Epkinly represents a significant milestone in transforming the treatment landscape for Diffuse Large B-cell Lymphoma (DLBCL). As the first and only bispecific antibody authorized for the treatment of adult patients with relapsed or refractory DLBCL in the United States, Epkinly showcases the biopharmaceutical industry’s commitment to translating cutting-edge scientific breakthroughs into practical and impactful medical solutions. Notably, Epkinly is the third FDA-approved treatment utilizing Genmab’s revolutionary DuoBody technology, highlighting the remarkable impact of this innovative approach in advancing patient care.
DLBCL is the most common form of Non-Hodgkin’s Lymphoma (NHL) worldwide, accounting for approximately 30 percent of all NHL cases. Despite recent treatment advancements, managing this rapidly growing cancer remains challenging, with 10%-15% of DLBCL patients experiencing refractory or relapsed disease, where cancer fails to respond to treatment. Epkinly belongs to a new class of cancer therapies known as bispecific CD20-directed CD3 T-cell engagers and is specifically designed to treat adults with relapsed or refractory DLBCL who have undergone at least two prior cancer therapies. Administered subcutaneously, Epkinly works by directing cytotoxic T-cells selectively to induce an immune response that destroys malignant cells. With the introduction of Epkinly, the medical community can aspire to enhanced therapies and improved prospects for individuals navigating the complexities of DLBCL.
5. Opvee Nasal Spray (nalmefene hydrochloride)
Treatment for: Opioid Overdose
Manufactured by: Opiant Pharmaceuticals, Inc.
Date of Approval: May 22, 2023
With the U.S. opioid crisis expanding to include synthetic opioids like fentanyl, which linger in the body for longer periods than traditional opioids, the National Institutes of Health (NIH) collaborated with pharmaceutical researchers and the U.S. government’s Biomedical Advanced Research and Development Authority to develop a drug that can rapidly revive users while providing protection against relapse. The newly approved drug Opvee is specifically designed to swiftly reverse the effects of an opioid overdose. It is formulated as a nasal spray version of the opioid antagonist Nalmefene, which was initially available as an injection in the 1990s. Nalmefene acts rapidly to reverse the dangerous effects of opioid overdose, such as respiratory depression, sedation, and low blood pressure, by blocking the opioid receptors in the brain and body. Opvee offers similar benefits to Naloxone, the life-saving drug widely used for decades. However, Opvee has longer-lasting effects, which reduces the risk of relapse in overdose patients. Indivior is expected to launch Opvee in October 2023.
6. Yuflyma Injection
Treatment for: Rheumatoid Arthritis, Juvenile Idiopathic Arthritis, Psoriatic Arthritis, Ankylosing Spondylitis, Crohn’s Disease, Ulcerative Colitis, Plaque Psoriasis, and Hidradenitis Suppurativa.
Company: Celltrion, Inc.
Date of Approval: May 23, 2023
Yuflyma, a biosimilar drug to Humira, is a tumor necrosis factor (TNF) blocker used to alleviate pain, inflammation, and other symptoms associated with various chronic autoimmune conditions by blocking the activity of TNF. TNF is a cytokine, which is a type of protein that triggers inflammation leading to autoimmune disorders. TNF blockers like Yuflyma provide relief from conditions such as rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, Crohn’s disease, ulcerative colitis, plaque psoriasis, and hidradenitis suppurativa. In the United States, more than 80% of individuals prescribed Humira rely on a high-concentration, citrate-free formulation of the medication. Yuflyma is available as a 40mg/0.4mL single-dose prefilled autoinjector and serves as a high-concentration, citrate-free alternative for patients seeking treatment options for their autoimmune conditions. Yuflyma is Celltrion’s fifth biosimilar and second anti-TNF biosimilar FDA-approved drug for use in the United States. It is expected to be available to patients in the country in July 2023.
7. Xacduro Kit for Injection (sulbactam and durlobactam (co-packaged)
Treatment for: Acinetobacter Pneumonia
Company: Innoviva, Inc.
Date of Approval: May 23, 2023
Xacduro, a co-packaged antibiotic injection, provides hope in the battle against hospital-acquired bacterial pneumonia (HABP) and ventilator-associated bacterial pneumonia (VABP) caused by susceptible strains of Acinetobacter baumannii-calcoaceticus complex. Acinetobacter infections pose a significant threat to human health, especially for individuals with compromised immune systems or those who are subjected to prolonged hospital stays. The World Health Organization has declared Acinetobacter a top critical threat to human health, and it is primarily spread through person-to-person contact or direct contact with contaminated surfaces. In the United States, more than 8,500 Acinetobacter infections are reported annually.
Xacduro has received FDA approval for use in adult patients aged 18 and older. It contains sulbactam and durlobactam, specifically targeting these challenging infections that often exhibit resistance to conventional antibiotics. The approval of Xacduro by the FDA emphasizes the urgent need for effective treatments for Acinetobacter-related pneumonia. Xacduro represents a promising leap forward in addressing this critical healthcare concern. Its introduction brings renewed optimism in the fight against Acinetobacter infections, equipping healthcare providers with a vital tool in their arsenal to combat these complex and dangerous pathogens.
8. Inpefa Tablets (sotagliflozin)
Treatment for: Heart Failure
Company: Lexicon Pharmaceuticals, Inc.
Date of Approval: May 26, 2023
Inpefa is an oral tablet taken once daily that offers the potential to reduce the risk of cardiovascular death, hospitalization for heart failure, and urgent heart failure visits in adults who have heart failure, type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The drug was discovered using Lexicon’s state-of-the-art approach to gene science. It acts as an inhibitor of both sodium-glucose co-transporter type 2 (SGLT2) and type 1 (SGLT1). The SGL inhibitor class was recommended by the American Heart Association (AHA), the American College of Cardiology (ACC), and the Heart Failure Society of America (HFSA) as a top-line treatment option for heart failure in their joint 2022 AHA/ACC/HFSA Guideline for the Management of Heart Failure. The approval of Inpefa was based on the positive outcomes observed in two large-scale phase III cardiovascular outcome studies, SOLOIST-WHF and SCORED, which included nearly 12,000 patients. These studies demonstrated that Inpefa effectively reduced the incidence of hospitalizations and urgent visits related to heart failure. Notably, investigations revealed a 33% reduction in the risk of cardiovascular death compared to a placebo among individuals recently hospitalized for worsening heart failure.
The prevalence of heart failure in the U.S. is projected to increase from 6.7 million to 8.0 million by 2030, highlighting it as a significant health concern, particularly among individuals aged 65 and older, who experience high rates of hospitalization. In addressing multiple underlying factors such as heart failure, type 2 diabetes mellitus, and chronic kidney disease, Inpefa recognizes the interconnected nature of these conditions and their impact on cardiovascular health. Lexicon expects Inpefa to be available by the end of June 2023. The company plans to set the medicine’s wholesale acquisition cost comparable to existing branded heart failure medications.
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