FDA goes on a rulings binge

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FDA ROUNDUP: In one of the busiest rulings binges of the year, the US Food and Drug Administration last week approved an assortment of pharmaceuticals, medical devices, an AI algorithm that analyzes chest X-rays, a smartphone-based urine test and a long-sought treatment for peanut allergy. But that’s just the beginning, the agency also warned e-cigarette companies about making false safety claims, cautioned about the possible severe lung infection in use of certain breast-cancer meds and began investigating widely used heartburn medicines likely containing a carcinogen.


But the beat goes on this week, beginning with our spotlight story. The FDA on Tuesday, September 17, 2019 announced Project Orbis, an initiative of the FDA Oncology Center of Excellence (OCE). Project Orbis provides a framework for concurrent submission and review of oncology drugs among its international partners. Under this project, the FDA, the Australian Therapeutic Goods Administration (TGA) and Health Canada collaboratively reviewed applications for two oncology drugs, allowing for simultaneous decisions in all three countries.

“We are pleased to be working alongside our Australian and Canadian colleagues to help make potentially life-changing treatments available to patients as quickly as possible while still ensuring the FDA’s high standards of safety and effectiveness,” said Acting FDA Commissioner Ned Sharpless, M.D. “As Project Orbis expands, we look forward to welcoming additional international partners to collaborate with us in this important initiative as we work to help further serve the global patient community.”

Collaboration among international regulators may allow patients with cancer to receive earlier access to products in other countries where there may be significant delays in regulatory submissions, regardless of whether the product has received FDA approval. This is partly due to different standards of care around the world that also have an impact on the increasingly international conduct of cancer clinical trials, potentially slowing the development of anticancer products. With a framework for concurrent submission and review of oncology drugs, Project Orbis facilitates a collaborative review to identify any regulatory divergence across review teams, the FDA’s Press Release reports.

As part of Project Orbis, in conjunction with decisions by TGA and Health Canada, the FDA on Tuesday granted accelerated approval to Lenvima (lenvatinib), from Eisai Co. Ltd., in combination with Keytruda (pembrolizumab), from Merck & Co. The approval is for the treatment of patients with advanced endometrial carcinoma that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) and who have disease progression following prior systemic therapy but are not candidates for curative surgery or radiation.


Here’s the regulatory action from Silver Spring, MD, for the week of September 9, 2019:

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx Inc. (Fremont CA) has won an approval from the FDA to market Ibsrela (tenapanor) for irritable bowel syndrome. The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase 3 trials.

Elsewhere, the FDA approved Aspen Pharmacare Holdings Ltd.’s hydroxyprogesterone caproate (HPC), which is used to prevent preterm birth in pregnant women, the Durban, South Africa-based drugmaker said. Aspen, which operates in about 56 countries, has said before that it views the US as presenting targeted growth opportunities for its niche women’s health products such as conjugated estrogens, which is used in menopausal hormone therapy. Aspen got the FDA abbreviated new drug application approval in early August for a preservative-free, single-dose vial.

The FDA on Friday expanded approval of Germany-based Boehringer Ingelheim GmbH’s Ofev (nintedanib) to include slowing the rate of decline in pulmonary function in adults with interstitial lung disease associated with systemic sclerosis (SSc-ILD). The US agency noted that Ofev is the first treatment approved by the FDA for this condition.

The FDA has cleared an artificial intelligence (AI) algorithm from GE Healthcare (Chicago) that analyzes chest x-rays for pneumothorax and helps flag suspected cases for radiologists to prioritize reading, the company announced. The algorithm, part of a set of other quality-assurance algorithms named the Critical Care Suite, was developed to run on a GE Healthcare mobile x-ray device.

The FDA has expanded the indication for mepolizumab (Nucala) from GlaxoSmithKline PLC (London) to children as young as 6 years old with severe eosinophilic asthma. Mepolizumab subcutaneous injection was first approved in the US in 2015 as an add-on maintenance treatment for patients with severe asthma who were aged 12 years or older and who had an eosinophilic phenotype.

The FDA approved Xeris Pharmaceuticals Inc.’s (Chicago) glucagon therapy that aims to treat severely low sugar levels in diabetes patients, the drugmaker said. The condition, called hypoglycemia, can cause acute cognitive impairment. A mild or moderate fall in sugar levels, when left untreated, can result in cardiovascular disease, seizure, coma and even death. Gvoke, Xeris’s glucagon pen is a pre-filled device with liquid stable form of glucagon. It won approval for use in diabetes patients aged 2 years and above.

Israel’s Healthy.io Ltd. (Tel Aviv) said on Thursday it received clearance from the FDA for its smartphone-based urine albumin test to aid in diagnosing chronic kidney disease. “The smartphone has the potential to be the great equalizer of health care,” said Yonatan Adiri, founder and CEO of Healthy.io. This is the company’s second FDA clearance. Last year it received clearance for its at-home, smartphone-based urinalysis test kit and now has a partnership with Boots UK, placing its test kits in hundreds of pharmacies.

The first-ever drug regimen to blunt acute peanut allergies in children should be approved, experts told an FDA committee on Friday, because the therapy has the potential to reduce the risk of life-threatening reactions and to improve patients’ lives. The new drug, called Palforzia and made by Aimmune Therapeutics Inc. (Brisbane CA), is an oral immunotherapy regimen that aims to reduce sensitivity to peanut allergens. It gradually exposes children to small amounts of peanut protein over the course of six months, until they can safely eat the equivalent of two peanuts.

Merck KGaA (Darmstadt DEU) received FDA “breakthrough” therapy status for tepotinib, a treatment for MET exon 14 skipping-mutated non-small cell lung cancer (NSCLC), joining Novartis AG’s capmatinib and a Pfizer Inc. treatment (Xalkori) in MET NSCLC drugs with “breakthrough” status. Although the announcement has been viewed by some as putting Merck KGaA on par with Novartis and Pfizer, the designation is only for treating patients whose cancers progressed following platinum chemotherapy. Both Novartis and Pfizer have had that designation for some time.

Investigators involved in developing a cancer drug viewed as a bright prospect in Amgen Inc.’s (Thousand Oaks CA) early-stage oncology pipeline have halted enrollment of new patients after signs of “cardiac toxicity” at a related drug with the same mechanism raised safety concerns at the FDA, prompting a clinical hold. The pharma giant noted in an oncology update that their dose-ranging study of the MCL1 inhibitor AMG 397 was placed on clinical hold by the agency, which prompted researchers to voluntarily halt enrollment in another early study of their other, more advanced, MCL1 program for AMG 176.

The FDA warned leading e-cigarette maker Juul Labs Inc. (San Francisco) about illegally marketing its product as a safer alternative to cigarettes. The FDA ordered Juul to respond within 15 working days with corrective actions and its plan to comply with federal law. The warning letter noted that failure to comply could result in fines, seizures or injunction.

The FDA is expected to approve Novo Nordisk AS’s (Bagsvaerd DNK) highly anticipated oral version of its Type 2 diabetes drug Ozempic (semaglutide) before the end of the year. But the Institute for Clinical and Economic Review (ICER) no longer waits for the price of a new drug to be announced before determining whether it will be cost effective–nor does the organization mince words when it makes a negative ruling. Oral Ozempic, ICER said in a draft report, only offers an “incremental benefit” in preventing adverse cardiac events when compared with products such as Jardiance from Eli Lilly and Boehringer Ingelheim. And because heart benefits comprise only one piece of the complicated treatment puzzle that each diabetes patient presents, “other treatments may provide better overall benefit and at lower costs.”

The FDA and European regulators are investigating medicines that contain ranitidine (multiple brands) for the presence of the probable carcinogen N-nitrosodimethylamine (NDMA), the same impurity that fueled a recall of numerous so-called sartan blood pressure and heart failure drugs last year. Ranitidine is an over-the-counter (OTC) and prescription histamine-2 blocker that reduces the production of stomach acid. OTC ranitidine is approved for prevention and relief of heartburn. Prescription ranitidine is approved for multiple indications, including treatment and prevention of ulcers of the stomach and intestines and treatment of gastroesophageal reflux disease.

And the FDA has issued a warning concerning the incidence of rare severe lung inflammation in some patients receiving Pfizer Inc.’s Ibrance (palbociclib), Novartis’s Kisqali (ribociclib) or Eli Lilly & Co.’s Verzenio (abemaciclib), all cyclin-dependent kinase 4/6 inhibitors that are used to treat breast cancer. Labeling has been updated about the risk. The agency recommends that patients talk to their doctors before considering stopping treatment.

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