LONDON (dpa-AFX) - As October draws to a close, it's time to take a look at the regulatory news that made headlines in the month and look ahead to what's in store for November.
Pfizer's Hympavzi received FDA approval on October 11, for the treatment of Hemophilia, which becomes the first of its kind to work by targeting a protein in the blood clotting process. Astellas followed suit on October 18, with Vyloy, the first CLDN18.2-targeting drug approved in the U.S. for specific gastric cancers. CLDN18.2 is a transmembrane protein. Iterum's Orlynvah received approval on October 25, marking a milestone as the first oral penem approved for use in the U.S. and the second FDA-approved treatment for uncomplicated urinary tract infections in the past two decades.
The FDA has approved 38 novel drugs so far this year (Jan - Oct) compared to 46 during the same time frame last year.
Now let's take a look at the drugs that are at the FDA altar, awaiting a decision in November.
Journey Medical Corp. (DERM)
The FDA decision on Journey Medical's DFD-29, proposed for the treatment of inflammatory lesions and erythema of rosacea in adults, is due on November 4, 2024.
In clinical settings, DFD-29 demonstrated statistical superiority over both the current standard-of-care treatment, Oracea 40 mg capsules, and placebo for Investigator's Global Assessment treatment success as well as the reduction in the total inflammatory lesion count.
If approved, DFD-29 would be the only oral, systemic therapy to address both inflammatory lesions and erythema (redness) from rosacea. The company expects DFD-29 to achieve peak annual net sales exceeding $100 million.
Journey Medical is a partner company of Fortress Biotech Inc. (FBIO).
DERM closed Tuesday's (Oct.29, 2024) trading at $5.74, down 4.33%.
Merus N.V. (MRUS)
Merus' lead drug candidate, Zenocutuzumab (Zeno), proposed for the treatment of patients with neuregulin 1 fusion (NRG1+) non-small cell lung and NRG1+ pancreatic cancer, is under priority review by the FDA.
NRG1 fusions are gene fusions regarded as oncogenic drivers and can be found in various tumor types.
The Biologics License Application for Zeno was accepted by the U.S. regulatory agency on May 6, 2024. Although a specific decision date hasn't been given, the application is under priority review, which usually means a decision will be made within six months. Therefore, we can expect the decision to be announced by November 6, 2024.
Zenocutuzumab has the potential to be the first and only targeted therapy for patients with NRG1+ lung and pancreatic cancer and may offer a substantial improvement over currently available therapies, according to the company.
MRUS closed Tuesday's trading at $51.92, down 1.70%.
Astellas Pharma Inc. (ALPMY.OB)
Astellas Pharma's supplemental New Drug Application for label update, seeking to include positive 2-year data for its marketed drug IZERVAY for the treatment of geographic atrophy secondary to age-related macular degeneration, awaits the FDA decision on November 19, 2024.
The sNDA was based on results from GATHER2, a phase III clinical trial that evaluated the efficacy and safety of monthly (EM) and every other month (EOM) dosing of IZERVAY through year 2. The GATHER2 data demonstrated that IZERVAY continued to reduce the rate of geographic atrophy lesion growth in patients with geographic atrophy secondary to age-related macular degeneration through 2 years versus sham.
IZERVAY was approved by the FDA on August 4, 2023. During the quarter from April 1, 2024, to June 30, 2024, the drug's sales totaled 12.7 billion yen.
ALPMY.OB closed Tuesday's trading at $11.64, up 3.39%.
PTC Therapeutics Inc. (PTCT)
PTC Therapeutics' Upstaza, a gene therapy for the treatment of AADC deficiency, is under FDA review, with a decision anticipated on November 13, 2024.
Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder that affects the brain. It is characterized by poor muscle tone (hypotonia), delayed development in head control, crawling, speech, Involuntary eye movements, abnormally excessive sweating (hyperhidrosis), Hypersalivation, Drooping eyelids (ptosis), gastrointestinal problems such as reflux, diarrhea, or constipation, behavioral problems, and sleep disturbances, among others.
Upstaza is a one-time gene replacement therapy that is proposed for the treatment of patients aged 18 months and older with a clinical, molecular, and genetically confirmed diagnosis of AADC deficiency with a severe phenotype. In clinical trials, Upstaza demonstrated transformational neurological improvements.
This gene therapy, directly administered into the brain, was authorized in the EU to treat adults and children aged 18 months and older with severe AADC deficiency with a genetically confirmed diagnosis, in July 2022, and reportedly carries a list price of about $3.7 million per dose.
PTCT closed Tuesday's trading at $42.50, up 4.99%.
Autolus Therapeutics plc (AUTL)
The FDA decision on Autolus Therapeutics' Obe-cel, proposed for the treatment of patients with relapsed/refractory adult B-cell acute lymphoblastic leukemia, is expected on November 16, 2024.
Obe-cel, also known as Obecabtagene autoleucel, is a CD19 CAR T cell investigational therapy designed to overcome the limitations in clinical activity and safety compared to current CD19 CAR T cell therapies like Bristol Myers' Breyanzi, Gilead's Tecartus and Yescarta, and Novartis' Kymriah.
In a pivotal study, the results of which formed the basis of the Biologics License Application, 78% of patients treated with Obe-cel achieved a complete response (CR) or CR with incomplete hematological recovery (CRi). Analysis of longer follow-up data from the pivotal study, announced in June 2024, showed that 40% of patients were in ongoing remission without subsequent stem cell transplant (SCT) or other intervention.
If approved, Obe-cel will have to compete with Amgen's Blincyto, which generated sales of $508 million in the first half of 2024, an increase of 27% over the year-ago period.
AUTL closed Tuesday's trading at $4.34, down 4.62%.
Applied Therapeutics Inc. (APLT)
Applied Therapeutics' Govorestat, proposed for the treatment of Classic Galactosemia, awaits the FDA decision on November 28, 2024.
Govorestat is a novel Aldose Reductase Inhibitor and a potent and selective compound, which crosses the blood-brain barrier into the Central Nervous System. Aldose Reductase plays a crucial role in the development of Galactosemia.
Galactosemia is a rare, genetic disorder where the body is unable to break down the sugar galactose. Instead, the galactose is converted into galactitol by the enzyme Aldose Reductase, leading to a buildup of this toxic metabolite in tissues and organs resulting in long-term disease complications. There is no known cure or approved treatment for Galactosemia. About 3,000 individuals in the US and 4,000 in Europe are affected by Galactosemia. Classic Galactosemia is the most common subtype of Galactosemia.
Govorestat is also under review by the EMA's Committee for Medicinal Products for Human Use (CHMP). A decision by the EMA is expected early in the first quarter of 2025.
Analysts at Leerink Partners expect Govorestat to achieve global peak sales of roughly $870 million in 2035, assuming it receives approval.
APLT closed Tuesday's trading at $8.75, up 1.39%.
BridgeBio Pharma Inc. (BBIO)
The FDA decision on BridgeBio Pharma's Acoramidis, an investigational drug for the treatment of transthyretin amyloid cardiomyopathy, is due on November 29, 2024.
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare but severe cause of restrictive cardiomyopathy caused by the accumulation of transthyretin fibrils in the myocardium. It can present with new or worsening heart failure or new conduction system disease. (Source: NIH).
In a phase III trial, dubbed ATTRibute-CM, Acoramidis treatment demonstrated an 81% absolute survival rate and a 0.29 observed mean annual cardiovascular-related hospitalization (CVH) frequency, as well as improvements for a large proportion of patients.
If approved, Acoramidis will have to compete with Pfizer's Vyndaqel and Vyndamax, the first treatments for ATTR-CM to receive FDA approval in May 2019. Acoramidis has the potential to hit blockbuster sales at its peak, according to analysts.
BBIO closed Tuesday's trading at $24.50, up 0.25%.
Jazz Pharmaceuticals plc (JAZZ)
The FDA decision on Zanidatamab, the human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody, for the treatment of previously treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer, is due on November 29, 2024.
Zanidatamab is being developed by Jazz Pharmaceuticals and BeiGene, Ltd. (BGNE) under license agreements from Zymeworks Inc. (ZYME), which first developed the molecule.
In the U.S., Europe, and Japan, around 12,000 individuals are diagnosed each year with HER2+ biliary tract cancer (BTC). If approved, Zanidatamab would be the first HER2-targeted therapy specifically for patients with locally advanced or metastatic HER2+ BTC.
According to Jazz Pharma, Zanidatamab could potentially reach peak sales of $2 billion.
JAZZ closed Tuesday's trading at $111.91, up 0.48%.
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