LONDON (dpa-AFX) - The FDA has approved 43 novel drugs so far this year (January-November), compared to 52 during the same period last year. As we step into the final month of the year, let's take a look at the biotech companies whose drugs are under regulatory review in the U.S., with crucial decisions expected in December.
BeiGene Ltd. (BGNE)
The FDA decision on BeiGene's Tevimbra in combination with fluoropyrimidine- and platinum-containing chemotherapy, for the treatment of patients with locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma is due in December.
Tevimbra, administered by intravenous infusion once every 3 weeks, is already approved in the U.S. for the treatment of adult patients with unresectable or metastatic esophageal squamous cell carcinoma after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor. Approved in March of this year, the drug was commercially launched in the U.S. in October.
The FDA is also reviewing Tevimbra as a first-line treatment for patients with unresectable, recurrent, locally advanced, or metastatic esophageal squamous cell carcinoma. Although the decision deadline passed in July 2024, no decision has been reached yet for this particular indication.
The drug is approved in the European Union and Australia for the treatment of metastatic non-squamous non-small cell lung cancer and squamous oesophageal cancer. In China, Tevimbra is approved for 13 indications.
Sales of Tevimbra were $163 million in the third quarter of 2024, representing growth of 13% compared to the prior-year period.
BGNE is currently trading at $211.01, down 1.86%.
Ionis Pharmaceuticals Inc. (IONS)
Ionis Pharma's investigational drug Olezarsen, proposed for the treatment of familial chylomicronemia syndrome, is under priority review by the FDA, with a decision due on December 19, 2024.
Familial Chylomicronemia Syndrome (FCS) is a rare genetic disorder characterized by extremely elevated triglyceride levels in the blood. This leads to a build-up of large triglyceride-containing particles in the blood called chylomicrons, which can result in severe health complications.
People with FCS are at high risk of acute pancreatitis, a painful condition where the pancreas becomes inflamed. They may also experience long-term health problems like fatigue and frequent, severe stomach pain.
Olezarsen is designed to lower the body's production of apoC-III, a protein produced in the liver that regulates triglyceride metabolism in the blood.
Currently, there are no FDA-approved drugs to treat FCS.
IONS is currently trading at $36.09, up 1.01%.
Lexicon Pharmaceuticals Inc. (LXRX)
The FDA decision on Lexicon Pharma's drug candidate Zynquista as an adjunct to insulin therapy for glycemic control in adults with type 1 diabetes and chronic kidney disease is expected on December 20, 2024.
Zynquista is an oral dual inhibitor of two proteins responsible for glucose regulation known as sodium-dependent glucose co-transporter types 1 and 2 (SGLT1 and SGLT2). SGLT1 is responsible for glucose absorption in the gastrointestinal tract, and SGLT2 is responsible for glucose reabsorption by the kidney.
This is Zynquista's second go-around with the U.S. regulatory agency. The FDA had declined to approve Zynquista in March 2019.
In October of this year, an FDA panel, that scrutinized Zynquista, voted against it, saying that the drug's benefits do not outweigh the risks.
Zynquista was granted marketing authorization in the European Union in April 2019 as an adjunct to insulin therapy to improve glycemic control in adults with type 1 diabetes mellitus. However, the European Commission withdrew the drug from the European Union in March 2022 at the request of Guidehouse Germany GmbH, the holder of Zynquista's marketing rights in Europe, which opted not to sell the product.
LXRX is currently trading at $0.79, down 0.84%.
AstraZeneca plc (AZN)
The FDA decision on Datopotamab deruxtecan, proposed for the treatment of adult patients with locally advanced or metastatic nonsquamous non-small cell lung cancer who have received prior systemic therapy, is due on December 20, 2024.
Datopotamab deruxtecan, a specifically engineered TROP2-directed DXd antibody-drug conjugate, is being jointly developed by Daiichi Sankyo and AstraZeneca. TROP2 is a protein broadly expressed in the majority of NSCLC tumours. There is currently no TROP2-directed ADC approved for the treatment of lung cancer.
In a phase III trial, Datopotamab deruxtecan showed a median overall survival of 14.6 months in a subgroup of patients with nonsquamous NSCLC while docetaxel, the current standard of care chemotherapy, demonstrated a median overall survival of 12.3 months.
If approved, Datopotamab deruxtecan could be the first TROP2-directed antibody-drug conjugate for patients with lung cancer.
AZN is currently trading at $66.98, down 0.95%.
Zealand Pharma A/S (ZEAL.CO)
Zealand Pharma's novel glucagon-like peptide-2 agonist, once-weekly Glepaglutide, proposed for the treatment of adults with short bowel syndrome dependent on parenteral support, is at the FDA altar, awaiting a decision on December 22, 2024.
Short bowel syndrome is a rare, chronic, and debilitating condition that leads to reduced ability of the intestines to absorb nutrients. It is associated with serious medical issues such as liver and kidney failure, metabolic disorders, chronic fatigue, and severe infections that can be life-threatening.
Shire's GATTEX is the first and only FDA-approved glucagon-like peptide-2 (GLP-2) analog for individuals aged 1 year and older with short bowel syndrome (SBS) who rely on parenteral support. The drug generated global annual sales of over $850 million in fiscal 2023.
Glepaglutide, if approved, can reduce both the burden of parenteral support and of daily dosing of the existing GLP-2 treatment (GATTEX) for people living with SBS and intestinal failure, according to the company.
Rhythm Pharmaceuticals Inc. (RYTM)
Rhythm Pharma has sought FDA approval to expand the use of IMCIVREE to include children as young as 2 years old for its approved indications, and a decision is due on December 26, 2024.
IMCIVREE, a melanocortin-4 receptor (MC4R) agonist, is already approved in the U.S. to treat adult and pediatric patients 6 years of age and older for chronic weight management of obesity caused by proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency, and Bardet-Biedl syndrome. The drug is also approved in Europe and the UK.
The label expansion to treat patients at an even earlier age will positively affect the lives of these children and their families, according to the company.
IMCIVREE generated global annual sales of $77.4 million in 2023 compared to $16.9 million in 2022.
RYTM is currently trading at $60.71, down 2.14%.
Soleno Therapeutics Inc. (SLNO)
Soleno's New Drug Application seeking approval of DCCR (diazoxide choline) for Prader-Willi syndrome in individuals four years and older who have hyperphagia is under Priority Review by the FDA, with a decision due on December 27, 2024.
The Prader-Willi Syndrome (PWS) is a rare genetic disorder with the hallmark symptom being hyperphagia, a chronic and life-threatening condition characterized by feelings of intense, persistent hunger, food pre-occupation, and an extreme drive to seek and consume food. It is also associated with behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development.
In a phase III trial setting involving PWS patients, DCCR showed promise in addressing hyperphagia, as well as several other symptoms such as aggressive/destructive behaviors, fat mass, and other metabolic parameters.
If approved, DCCR would be the first treatment for Prader-Willi syndrome with hyperphagia.
According to Robert W. Baird analyst, Brian Skorney, DCCR could generate over $800 million in peak U.S. sales, with the potential to exceed $1 billion globally after approvals.
SLNO is currently trading at $50.99, down 3.26%.
Checkpoint Therapeutics Inc. (CKPT)
Checkpoint Therapeutics' investigational drug, Cosibelimab, is back at the FDA altar, awaiting a decision on December 28, 2024.
Cosibelimab, an anti-programmed death ligand-1 ('PD-L1') antibody, is proposed for the treatment of adults with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or curative radiation.
This is the company's second attempt to secure FDA approval for Cosibelimab. The U.S. regulatory agency had declined to approve Cosibelimab last December mainly due to inspection findings at a third-party manufacturer. No concerns about the clinical data package, safety, or labeling for the approvability of Cosibelimab were raised then, according to the company.
Checkpoint was founded by Fortress Biotech, Inc. (FBIO).
Cutaneous squamous cell carcinoma (cSCC) is the second most common type of skin cancer, with more than 1 million cases diagnosed annually in the U.S. The U.S. cSCC market opportunity is valued at over $1 billion.
Will Cosi get a piece of the pie in the blockbuster market opportunity?
CKPT is currently trading at $4.34, down 1.14%.
Mirum Pharmaceuticals Inc. (MIRM)
Mirum Pharma's Chenodiol, proposed for the treatment of patients with cerebrotendinous xanthomatosis, is under FDA review, with a decision anticipated on December 28, 2024.
Cerebrotendinous xanthomatosis (CTX) is a rare, progressive inherited disorder, where the body cannot break down cholesterol properly - causing toxins (e.g., cholestanol and bile alcohols) to build up throughout the body over time. The signs and symptoms of CTX include neonatal cholestasis (jaundice or bile flow interruption), chronic diarrhea, the development of bilateral cataracts before the age of 18, development of tendon xanthomas (fatty deposits in the tendons) during teenage years or later, and neurologic deterioration.
Chenodiol, originally developed by Travere Therapeutics Inc. (TVTX), was acquired by Mirum when Travere sold its bile acid product portfolio to Mirum last year. The drug is approved in the U.S. for the treatment of people with radiolucent stones in the gallbladder.
MIRM is currently trading at $45.07, down 2.49%.
Bristol-Myers Squibb Company (BMY)
The FDA decision on Bristol Myers Squibb's (BMY) Biologics License Application for the subcutaneous formulation of Opdivo co-formulated with Halozyme's proprietary recombinant human hyaluronidase across all previously approved adult, solid tumor Opdivo indications is expected on December 29, 2024.
Opdivo is approved as monotherapy, monotherapy maintenance following completion of Opdivo plus Yervoy combination therapy, or in combination with chemotherapy or cabozantinib.
The currently approved formulation of Opdivo is intravenous administration and is indicated for the treatment of melanoma, non-small cell lung cancer, malignant pleural mesothelioma, renal cell carcinoma, classical Hodgkin lymphoma, squamous cell carcinoma of the head and neck, urothelial carcinoma, MSI-H or dMMR metastatic colorectal cancer, hepatocellular carcinoma, esophageal cancer, gastric cancer, and gastroesophageal junction cancer.
Opdivo generated global sales of $2.36 billion in the third quarter of 2024, an increase of 4% over the year-ago quarter.
BMY is currently trading at $59.16, down 0.10%.
Neurocrine Biosciences Inc. (NBIX)
Neurocrine Biosciences has two New Drug Applications for Crinecerfont under priority review by the FDA, with decisions expected on December 29 and December 30, 2024.
Crinecerfont, an investigational, oral, selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist being developed to reduce and control excess adrenocorticotropic hormone (ACTH) and adrenal androgens, is proposed for the treatment of children, adolescents, and adults with classic congenital adrenal hyperplasia.
Congenital adrenal hyperplasia (CAH) is a rare genetic condition that results in deficiency of the enzyme 21-hydroxylase (21-OHD) that alters the production of adrenal hormones which are essential for life. If left untreated, CAH can result in salt wasting, dehydration, and even death.
The company has sought approval for Crinecerfont as a capsule formulation and as an oral solution formulation.
If approved, Crinecerfont would be the first new treatment option for CAH in 70 years. Crinecerfont has the potential to become a blockbuster, according to analysts.
NBIX is currently trading at $127.59, up 0.66%.
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