As the calendar turns to 2024, the pharmaceutical world is on the threshold of a notable era, brimming with innovation and new hope for solutions for the woes of humanity. This article explores the essence of these developments by providing a thorough analysis of the most recent achievements in medical research.
The list of new FDA approved drugs & devices and those expected to get approval in 2024 opens up new avenues for medical advancements. These approvals ranging from ground-breaking cancer treatments to innovative cures for chronic ailments represent rays of hope in our never-ending fight against diseases. It’s critical to comprehend the underlying trends and technological advancements driving these breakthroughs as we explore through these innovations.
This path towards new FDA approved drugs & devices is evidence of the inventiveness of the researchers and their unwavering quest of universally improved health. Come along as we explore the complexities of some of these medical wonders and their enormous influence on healthcare systems, patient care, and the global society.
Spotlight on the New FDA Approved Drugs & Devices
Toripalimab (cancer treatment)
The FDA approved Shanghai Junshi Biosciences’s Toripalimab-tpzi on October 27, 2023 to treat nasopharyngeal carcinoma. Nasopharyngeal carcinoma is a type of head and neck cancer. Toripalimab is approved to be used along with cisplatin and gemcitabine hydrochloride to treat patients with recurrent nasopharyngeal carcinoma which has spread to the tissues nearby or other parts of the body.
Toripalimab’s effectiveness and safety continue was evaluated in numerous clinical trials such as JUPITER-02 and POLARIS-02, positioning it as a potential key player in the future of cancer therapy.
The JUPITER-02 trial proved that the combination of toripalimab with chemotherapy decreased the risk of development of cancer or death by around 50% in patients with recurrent or metastatic nasopharyngeal carcinoma.
While it is approved for first-line treatment of nasopharyngeal carcinoma, it also given approval for second-line treatment for melanoma and urothelial carcinoma. This development marks a significant step forward in cancer treatment, offering new hope for patients with these challenging conditions.
Zepbound (weight loss drug)
On November 8, 2023, the FDA had approved Eli Lily’s Zepbound (tirzepatide) for treating chronic obesity in adults. It is advised to follow strict low calorie diet and exercise in addition to the administration of the drug for better results. This weekly injected drug can activate GLP 1 receptors and reduce appetite and control the food intake.
Eli Lilly’s Mounjaro with the same tirzepatide, is approved for treating Type-2 diabetes but also used off-label for weight loss. However, in the recent past Eli Lilly is facing a lot of Mounjaro lawsuits for causing stomach paralysis.
The fact that Zepbound has the same problematic compound causing stomach paralysis in the users is raising questions on the efficacy and safety of the drug. However, Eli Lilly has not yet added any specific stomach paralysis related warning in the label.
The safety label only warns people with a family history of thyroid cancer (MTC) or MEN 2 endocrine condition, not to use the drug.
Other adverse Zepbound side effects mentioned in Eli Lilly’s website are listed below:
- Stomach problems
- Gallbladder problems
- Kidney failure
- Pancreatitis
- Allergic reactions
- Suicidal thoughts
- Change in vision in type 2 diabetic patients
Will Zepbound create fatal side effects like its counterparts? We have to wait and see the response of the users to know the efficacy and the potential adverse side effects.
iDose TR (travoprost intracameral implant)
On Dec 14, 2023, the FDA approved iDose TR (travoprost intracameral implant) by Glaukos Corporation, an ophthalmic technology and pharmaceutical company. This tiny implant for the eye reduces the intraocular pressure (IOP) in patients with ocular hypertension (OHT)/ open-angle glaucoma (OAG).
Once implanted, it would continuously deliver “therapeutic levels of a proprietary formulation of travoprost inside the eye for extended periods of time” round the clock.
The approval was given based on the results of the phase 3 trials, which proved that it is safe and efficient to use. More than 80% of the patients treated with the implant did not need any topical drops and more than 98% of the patients successfully continued the trial without any side effects.
This novel therapy is expected to relieve the patients from using eye drops for longer periods. Glaukos has planned to launch eye implant in the first quarter of 2024.
Zelsuvmi (Berdazimer)
Zelsuvmi, a topical antiviral gel, is approved by the FDA on January 5, 2024 for treating molluscum contagiosum, a common skin infection mostly found in children and rarely in adults. It is the first-in-class medication for treating this viral skin infection.
Molluscum contagiosum appears as painless small red bumps depressed in the center, which might be itchy. If scratched, the infection would spread to other parts of the body or to other people through contamination.
Zelsuvmi (Berdazimer gel 10.3%) is manufactured by Ligand Pharmaceuticals Incorporated (Novan Pharmaceuticals). It releases nitric oxide and provides an antimicrobial effect upon application.
The FDA has approved Zelsuvmi based on the pre-market clinical trials conducted on children. The study revealed that around 32.4% of children with molluscum were cured of the infection completely in 12 weeks.
Experts like Mark D. Kaufmann, MD, FAAD (Clinical Professor of Dermatology at Mount Sinai feel that it is a breakthrough invention allowing the doctors treat patients at home as it can be applied by the patient or a family member.
Zolbetuximab (Gastric Cancer)
Zolbetuximab from Astellas Pharma Inc. is given the PDUFA date on January 12, 2024 by the FDA. Zolbetuximab is designed for the first-line treatments for patients with Gastric and Gastroesophageal Junction adenocarcinoma and it would be the first-in-class CLDN18.2-targeted treatment in the U.S. The review would be based on the Spotlight and Glow clinical trials, which proved significant improvement in the patients when compared to their placebo drugs.
Dihydroergotamine (DHE) Nasal Powder (Acute Migraine)
Satsuma Pharmaceuticals’ dihydroergotamine (DHE) nasal powder product is accepted for review on January 17, 2024 (PDUFA date) for the treatment of acute migraine. This dry powder (DHE) would give faster absorption and best efficacy when compared to the existing nasal sprays. The ASCEND and SUMMIT trials proved better results in the most severe migraine symptoms.
Intranasal Scopolamine (motion sickness)
Intranasal Scopolamine Nasal Gel from NASA and Defender Pharmaceuticals gets the PDUFA date on January 26, 2024. This anti-nausea gel will be helpful to people with motion sickness by preventing nausea and vomiting. In the clinical trials, the participants would be subjected to ocean voyage set up and astronauts and ground-control people on provocative motion set ups. The rapid absorption of the drug is expected to provide enhanced results.
Low Dose Atropine 0.01% (Children with Myopia)
Vyluma’s low dose atropine 0.01% receives the PDUFA date on January 31, 2024. This drug is designed to address the issues of children with Myopia. The four year clinical trials proved safety and efficacy of the drug. If approved it would be the first-in-class treatment for the children with Myopia.
Lifileucel
Lifileucel is a new therapy invented by Iovance Biotherapeutics for treating advanced form if melanoma. It is first ever form of cell therapy for advanced melanoma. This therapy is under the FDA for review and approval. FDA has set a Prescription Drug User Fee Act target action date on February 24, 2024 as the FDA resources were insufficient to complete a quick review.
The clinical trial conducted on patients with advanced melanoma proved that it is beneficial for them. Lifileucel therapy is expected to be approved by the FDA in 2024 itself.
Elevidys (First gene therapy for DMD)
Sarepta Therapeutics, Inc. got the FDA’s accelerated approval for their Elevidys (delandistrogene moxeparvovec –rokl) as the first gene therapy to treat Duchenne muscular dystrophy (DMD) on June 22, 2023.
It is a single intravenous dose recombinant gene therapy in which a gene was injected into the body to stimulate the production of micro-dystrophin. It is a shortened version of the normal protein found in the muscle cells.
Duchenne Muscular Dystrophy (DMD) is a very rare genetic condition affecting the muscles. It causes weakening and wasting away of body muscles. The mutated genes does not contain dystrophin that maintains the body muscles.
The FDA approved the drug based on the two-part trial results submitted by the manufacturer showing benefits in 4 to 5 year old patients. They considered the risks of the drug, the nature of the disease in children and the urgent need of DMD drug.
However, Sarepta is asked by the FDA to submit a final report of the trial comparing Elevidys to “placebo in 125 ambulatory patients with DMD with confirmed mutation in the DMD gene” by January 31, 2024.
The common side effects reported of this therapy are noted below:
- Acute liver injury
- Elevation of troponin-I (a heart protein)
- Immune-mediated myositis Nausea
- Myocarditis
- Pyrexia (fever)
- Thrombocytopenia (abnormal low platelet count)
- Vomiting
Fordadistrogene movaparvovec
Fordadistrogene movaparvovec is another single-infusion gene therapy designed to treat Duchenne Muscular Dystrophy (DMD) by introducing a functional dystrophin gene to patients’ muscle cells, potentially addressing the genetic root of the disease.
Pfizer’s Fordadistrogene movaparvovec is undergoing Phase III trial to evaluate the efficacy and safety of the drug in boys with DMD at present and awaits approval from the FDA. Pfizer expects to get the approval by the second half of 2024.
Earlier a clinical hold was imposed on this therapy in December 2021 after the death of a patient in Phase 1b trial and it was lifted on April 2022.
Resmetirom (Nonalcoholic Steatohepatitis (NASH) drug)
Resmetirom is a thyroid hormone receptor-beta agonist designed to treat adult patients with liver fibrosis. It regulates fat metabolism and liver inflammation. It has been shown to reduce LDL-C and hepatic fat significantly over 52 weeks compared to a placebo.
FDA has set a review date for Resmetirom to treat people with nonalcoholic steatohepatitis (NASH) and nonalcoholic fatty liver. FDA has granted priority review and set a Prescription Drug User Fee Act (PDUFA) date on March 14, 2024. It’s manufacturer, Madrigal Pharmaceuticals hopes to market the drug in 2024.
Crovalimab
Crovalimab is yet another novel monoclonal antibody designed for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). PNH is a life threatening blood disease, which can destroy red blood cells, cause blood clots and bone marrow dysfunction. In September 2023, the FDA accepted the Biologics License Application (BLA) for this drug.
Designed by Chugai Pharmaceutical Co., Ltd, Crovalimab addresses various autoimmune and inflammatory diseases in people. Its targeted approach allows Crovalimab to potentially provide therapeutic benefits while minimizing unwanted side effects typically associated with broader immune suppression. The development of Crovalimab marks a significant step in personalized medicine, offering new hope for patients suffering from conditions that have historically been challenging to manage.
Crovalimab is expected to be approved by the FDA in 2024 and if approved it would be the first subcutaneous injection to be administered monthly to treat this condition.
Sotatercept
Sotatercept is a yet-to-be approved therapeutic drug formed to treat patients with pulmonary arterial hypertension (PAH), a severe and progressive disorder characterized by high blood pressure in the arteries of the lungs.
Designed by Merck, Sotatercept aims to reverse the vascular remodeling in the lungs, thereby alleviating the high pulmonary pressures and improving cardiac output. Functioning as a fusion protein, Sotatercept combines a portion of the human activin receptor type IIA with a fragment of a human immunoglobulin G1.
The FDA has accepted to do a priority review of BLA for this drug in September 2023 and it has allotted a Prescription Drug User Fee Act (PDUFA) date on March 26, 2024.
According to Dr. Joerg Koglin, senior vice president of Merck Research Laboratories,
The FDA’s acceptance of this application is an exciting milestone in our journey to bring this novel activin signaling inhibitor to patients. Based on the profound improvements across primary and secondary outcome measures in the Phase 3 STELLAR trial, we believe sotatercept has the potential to transform the treatment of patients with PAH. We look forward to working closely with the FDA to bring sotatercept to patients in need.
If approved, its innovative approach will offer hope for significant therapeutic advancements in the management of PAH, a condition where limited treatment options currently exist. Clinical trials have been focused on evaluating the safety, tolerability, and efficacy of Sotatercept, with promising results suggesting a potential breakthrough in PAH therapy.
Atidarsagene autotemcel (Libmeldy)
Orchard Therapeutics has received the FDA’s nod for priority review of BLA for their innovative drug, Libmeldy (Atidarsagene autotemcel) in September 2023. Libmeldy is a stem cell gene therapy designed to treat Metachromatic Leukodystrophy (MLD).
Metachromatic Leukodystrophy (MLD) is genetic disorder that leads to fat buildup in cells in brain, spinal cord, and peripheral nerves. This occurs due to the lack of enzyme which helps in the sulfatide (fat) break down. Sulfatide is needed in the maintenance of the myelin sheath. However, unusual buildup of sulfatide can destroy the myelin sheath. This in turn leads to the dysfunction of brain or peripheral nerves.
Libmeldy is already approved in U.K and Europe. Whereas in U.S., Libmeldy (OTL-200) is allotted a PDUFA date on March 18, 2024. The result of the clinical trials proved that Libmeldy could preserve motor function and cognitive development in the people involved in the study. It is hoped that based on these reports, Libmeldy will successfully secure the FDA approval in 2024 itself.
We have discussed only a handful of drugs and devices approved or waiting for approval in the recent future in this article. There are a few more in the list. We will discuss them in our future articles.
To wrap up,
Most of the new FDA approved drugs or those waiting to get approvals in 2024 are first-in-the-class drugs or devices that can bring a revolutionary change in the healthcare field and patient care. The drugs, which are well researched, carefully manufactured and diligently tested before marketing would bring most of the benefits they intended to provide to humankind. This would also help in the evolution of more innovative medical supplies in the future.
However, let’s not forget the fact that rushing defective medicines to the market purely to reap the monetary benefits compromising patient health would always risk the lives of common people and turn out to be detrimental to the future of the drug and the financial stability of pharmaceutical companies. The recent lawsuits on Wegovy, Ozempic, Nexium, Truvada and other similar pharma lawsuits hint at that.
Let’s hope these new medical marvels create significant and positive outcome in the healthcare field unlike the defective medicines we have seen in the past.
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