The Food and Drug Administration (FDA) has authorized 43 new drugs from January to November this year, a slight decrease from the 52 approvals in the same timeframe last year. As we enter the year's closing month, let's examine the biotechnology firms whose drugs are pending FDA evaluation in the United States, with pivotal decisions anticipated in December.
**BeiGene Ltd. (BGNE)**
A decision from the FDA regarding BeiGene's Tevimbra, when used with fluoropyrimidine- and platinum-based chemotherapy, is expected this December for patients with locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma.
Tevimbra, delivered via intravenous infusion every three weeks, already holds U.S. approval for adults with unresectable or metastatic esophageal squamous cell carcinoma following systemic chemotherapy that excluded PD-(L)1 inhibitors. Granted approval in March, it launched commercially in October in the U.S.
Additionally, Tevimbra is under FDA review as a first-line treatment for unresectable, recurrent, locally advanced, or metastatic esophageal squamous cell carcinoma. Despite a passed decision deadline in July 2024, a verdict is yet to be reached for this indication.
Globally, the drug is approved within the European Union and Australia for treating metastatic non-squamous non-small cell lung cancer and squamous esophageal cancer. In China, it is sanctioned for 13 different uses.
Tevimbra's sales reached $163 million in Q3 2024, marking a 13% increase from the same quarter last year. Currently, BGNE's shares are trading at $211.01, reflecting a decline of 1.86%.
**Ionis Pharmaceuticals Inc. (IONS)**
Ionis Pharmaceuticals' investigational treatment, Olezarsen, is under priority review by the FDA for familial chylomicronemia syndrome, with a decision expected by December 19, 2024.
Familial Chylomicronemia Syndrome (FCS) is a rare genetic disorder resulting in extremely high triglyceride levels, causing large triglyceride-rich particles, called chylomicrons, to accumulate in the bloodstream. This can lead to severe health issues.
FCS patients face notable risk for acute pancreatitis, an agonizing inflammatory condition of the pancreas, alongside long-term complications like fatigue and chronic abdominal pain.
Olezarsen aims to lower the synthesis of apoC-III, a liver-produced protein, instrumental in managing blood triglyceride metabolism. Presently, no FDA-approved drugs are available for FCS treatment.
Trading at $36.09, Ionis Pharmaceuticals' stock is witnessing a rise of 1.01%.
**Lexicon Pharmaceuticals Inc. (LXRX)**
Anticipation surrounds the FDA's impending decision regarding Lexicon Pharma's drug candidate, Zynquista, as an adjunctive therapy to insulin for adults managing type 1 diabetes with chronic kidney disease. The decision is expected on December 20, 2024.
Zynquista is an oral dual inhibitor targeting proteins SGLT1 and SGLT2, responsible for glucose absorption in the gastrointestinal tract, and renal glucose reabsorption, respectively.
This marks Zynquista's second assessment by U.S. regulators, following a non-approval in March 2019. A recent FDA panel review in October concluded that the drug's benefits do not compensate for its associated risks.
While Zynquista received European Union marketing authorization in April 2019 for type 1 diabetes control, the European Commission rescinded its approval in March 2022 after Guidehouse Germany GmbH opted against its distribution.
Lexicon's shares are currently priced at $0.79, reflecting a 0.84% decrease.
**AstraZeneca plc (AZN)**
AstraZeneca's awaited FDA verdict on Datopotamab deruxtecan is set for December 20, 2024. This treatment is intended for adults with locally advanced or metastatic nonsquamous non-small cell lung cancer following prior systemic therapies.
Datopotamab deruxtecan, a TROP2-directed DXd antibody-drug conjugate, is jointly developed by Daiichi Sankyo and AstraZeneca. TROP2, a prevalent protein in numerous NSCLC tumors, currently lacks an FDA-approved ADC treatment for lung cancer.
Clinical trials revealed that Datopotamab deruxtecan achieved a median overall survival of 14.6 months in nonsquamous NSCLC patients, compared with 12.3 months for docetaxel, the existing standard chemotherapy.If Datopotamab deruxtecan receives approval, it would represent the pioneering TROP2-directed antibody-drug conjugate specifically for lung cancer patients.
As of the latest trading, AstraZeneca (AZN) is priced at $66.98, marking a decrease of 0.95%.
**Zealand Pharma A/S (ZEAL.CO)**
Zealand Pharma is awaiting an FDA decision on its innovative glucagon-like peptide-2 agonist, once-weekly Glepaglutide, positioned for adults with short bowel syndrome reliant on parenteral support. The verdict is expected on December 22, 2024.
Short bowel syndrome is a rare, chronic ailment that significantly diminishes intestinal nutrient absorption capability. It is linked to severe health complications, including liver and kidney failure, metabolic disorders, chronic fatigue, and severe infections that threaten life.
Notably, Shire's GATTEX stands as the first and sole FDA-approved glucagon-like peptide-2 (GLP-2) analog for individuals aged 1 and older with short bowel syndrome that require parenteral support. The drug achieved over $850 million in global annual sales for fiscal 2023.
Upon approval, Glepaglutide may alleviate the burdens associated with parenteral support and the daily administration of the current GLP-2 therapy (GATTEX) for those with SBS and intestinal failure, as stated by the company.
**Rhythm Pharmaceuticals Inc. (RYTM)**
Rhythm Pharmaceuticals is pursuing expansion approval from the FDA to broaden the use of IMCIVREE to include children as young as 2 years for its established indications, with a decision due by December 26, 2024.
IMCIVREE, a melanocortin-4 receptor (MC4R) agonist, is already sanctioned in the U.S. for treating both adults and children from 6 years onwards for chronic weight management of obesity due to proopiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency, including Bardet-Biedl syndrome. Approvals also extend to Europe and the UK.
The proposed label expansion to accommodate younger patients is projected to significantly enhance the quality of life for affected children and their families, according to the company.
In 2023, IMCIVREE achieved global annual sales of $77.4 million, up from $16.9 million in 2022.
Currently, Rhythm Pharmaceuticals (RYTM) trades at $60.71, reflecting a 2.14% decline.
**Soleno Therapeutics Inc. (SLNO)**
Soleno is seeking FDA Priority Review approval for DCCR (diazoxide choline) as a treatment for Prader-Willi syndrome in individuals aged four and above with hyperphagia. A decision is anticipated by December 27, 2024.
Prader-Willi Syndrome is a rare genetic condition marked by hyperphagia, signified by chronic, intense hunger, pre-occupation with food, and an overwhelming drive to consume food. It is associated with behavioral challenges, cognitive impairments, reduced muscle tone, short stature when not treated with growth hormone, excess body fat, developmental delays, and incomplete sexual maturation.
In Phase III trials involving Prader-Willi Syndrome patients, DCCR demonstrated potential in alleviating hyperphagia and associated symptoms, including aggressive behaviors, fat mass, and metabolic parameters.
Upon approval, DCCR would be the inaugural treatment for hyperphagia in Prader-Willi Syndrome.
According to Robert W. Baird analyst Brian Skorney, DCCR could reach over $800 million in peak U.S. sales, with potential global sales exceeding $1 billion post-approval.
Soleno Therapeutics (SLNO) is presently trading at $50.99, down 3.26%.
**Checkpoint Therapeutics Inc. (CKPT)**
Checkpoint Therapeutics is seeking FDA approval for Cosibelimab, with a decision anticipated on December 28, 2024.
Cosibelimab, an anti-programmed death ligand-1 ("PD-L1") antibody, is targeted for adults with metastatic or locally advanced cutaneous squamous cell carcinoma, not candidates for curative surgery or radiation.
This constitutes the company's second submission for FDA approval of Cosibelimab. The FDA had previously rejected approval last December due to third-party manufacturer inspection findings. Nonetheless, the company asserts that no issues were identified concerning Cosibelimab's clinical data package, safety, or labeling.
Checkpoint, a Fortress Biotech, Inc. (FBIO) initiative, aims to penetrate the expansive market opportunity for cutaneous squamous cell carcinoma, which annually sees over 1 million new cases in the U.S., valuing the market at over $1 billion.
Checkpoint Therapeutics (CKPT) is trading at $4.34, showing a 1.14% decline.
**Mirum Pharmaceuticals Inc. (MIRM)**
Further information about Mirum Pharmaceuticals Inc. (MIRM) was not included in the draft pasted above. Mirum Pharma's Chenodiol, developed for the treatment of cerebrotendinous xanthomatosis (CTX), is currently under review by the FDA, with a decision expected by December 28, 2024. CTX is a rare genetic disorder characterized by the body’s inability to properly metabolize cholesterol, leading to the accumulation of toxins such as cholestanol and bile alcohols. Symptoms include neonatal cholestasis (jaundice or bile flow disruption), chronic diarrhea, early-onset bilateral cataracts, tendon xanthomas (fatty deposits in tendons) emerging during adolescence or later, and progressive neurological decline.
Chenodiol was originally developed by Travere Therapeutics Inc. before Mirum acquired the bile acid product portfolio from Travere last year. The medication is already approved in the U.S. for treating individuals with radiolucent gallstones. As of now, Mirum’s stock is trading at $45.07, reflecting a drop of 2.49%.
Turning to Bristol-Myers Squibb Company (BMY), the FDA's decision regarding their Biologics License Application for a subcutaneous formulation of Opdivo, which incorporates Halozyme's recombinant human hyaluronidase, is anticipated on December 29, 2024. Opdivo is approved for use as a standalone therapy, maintenance therapy after Opdivo plus Yervoy combination treatment, or in combination with chemotherapy or cabozantinib. The existing intravenous formulation of Opdivo treats various cancers, including melanoma, non-small cell lung cancer, malignant pleural mesothelioma, and several others.
Opdivo reported global sales of $2.36 billion for the third quarter of 2024, marking a 4% increase compared to the same period the previous year. Bristol-Myers Squibb stock currently trades at $59.16, down slightly by 0.10%.
In the case of Neurocrine Biosciences Inc. (NBIX), two New Drug Applications for Crinecerfont are under priority FDA review, with decisions expected on December 29 and December 30, 2024. Crinecerfont is an investigational oral selective CRF1 receptor antagonist developed to manage excessive levels of adrenocorticotropic hormone (ACTH) and adrenal androgens in patients with classic congenital adrenal hyperplasia (CAH).
CAH is a rare genetic disorder caused by a deficiency of the enzyme 21-hydroxylase, disrupting essential adrenal hormone production, which can lead to severe consequences if left untreated. Neurocrine has submitted Crinecerfont for approval in both capsule and oral solution forms. If approved, Crinecerfont would be the first new CAH treatment option in 70 years, with significant potential to become a blockbuster, as analysts suggest. Neurocrine Biosciences’ stock is currently trading at $127.59, representing a 0.66% increase.
