Sanofi, Pfizer & Bristol Drugs Among the 12 Approvals to Look Out for in Q4

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historical data from Yescarta’s ZUMA-1 trial and Kymriah’s JULIET trial, which reported CR rates of 51% and 32%, respectively. Only six patients (2%) experienced grade 3/4 cytokine release syndrome with Breyanzi, compared with 13% with Yescarta and 23% with Kymriah. SC141322

Kala Pharmaceuticals’ Eysuvis
Indication: Dry Eye

PDUFA action date: 30 October NDA – Second Review

Following a complete response letter in August 2019, Kala Pharmaceuticals, Inc. is hoping for better luck with this second review for Eysuvis (loteprednol etabonate ophthalmic suspension) for the short-term treatment of the signs and symptoms of dry eye disease. (Also see “Kala Needs Big STRIDE To Get Dry Eye Drug OK” – Scrip, 12 Aug, 2019.)

Currently Eysuvis is positioned to be a short-term treatment of dry eye disease rather than as a maintenance therapy. Since Novartis’ Xiidra (lifitegrast) and C#11116:Allergan AG]’s Restasis (cyclosporine ophthalmic emulsion) are both used as long-term treatments, this should allow Eysuvis to differentiate itself within this competitive indication.

The CRL indicated that efficacy data from an additional clinical trial would be needed to support a resubmission, which led to the Phase III STRIDE 3 trial to supplement the previously completed Phase II trial and the first two Phase III efficacy and safety trials, STRIDE 1 and STRIDE 2. (Also see “Kala’s Third Phase III Trial Should Line Up Approval In Dry Eye” – Scrip, 9 Mar, 2020.)

Kala resubmitted its application in April with the STRIDE 3 trial data and this was accepted as a Class 2 response with a PDUFA target action date of 30 October. The study met both of its primary symptom endpoints, showing a significant improvement in ocular discomfort severity in both the overall ITT population and in a predefined subgroup of ITT patients with more severe ocular discomfort at baseline.

Regeneron’s REGN-EB3
Indication: Ebola

PDUFA action date: 23 October BLA – First Review

If approved, Regeneron Pharmaceuticals, Inc.’s Ebola therapy REGN-EB3 would be a positive example of how synergy between a government agency, the Biomedical Advanced Research and Development Authority (BARDA) of the US Department of Health and Human Services (HHS) and a biopharmaceutical company can expedite the drug development process, analysts at Biomedtracker say.

The product, a cocktail of three human IgG1 mAbs (REGN3470, REGN3471, and REGN3479) directed against different epitopes on the Ebola virus glycoprotein, was developed under an agreement announced in 2015 between the two parties for the treatment of Ebola virus infection.

Regeneron initiated a rolling BLA submission for REGN-EB3 for the treatment of Ebola virus infection based on the pivotal Phase II PALM trial, which tested four potential therapies for Ebola: REGN-EB3, Mapp Biopharmaceutical Inc.’s ZMapp and mAb114 (a single monoclonal antibody which was developed by scientists at the US NIH’s National Institute of Allergy and Infectious Diseases); and one small-molecule antiviral, Gilead’s remdesivir. (Also see “Ebola Success For Regeneron’s Triple Antibody Cocktail ” – Scrip, 13 Aug, 2019.)

[Editor’s note: The FDA approved Regeneron’s Ebola drug on Oct. 14.]

Eiger’s Zokinvy
Indication: Hutchinson-Gilford Progeria Syndrome

PDUFA action date: 20 November NDA – First Review

Having previously failed in treating myelodysplastic syndrome (MDS), breast cancer, brain cancer, and non-small cell lung cancer, an approval for Eiger BioPharmaceuticals, Inc.’s Zokinvy (lonafarnib) for Hutchinson–Gilford progeria syndrome the (HGPS) would be the breakthrough the drug needs, say Biomedtracker analysts. (Also see “Eiger Shifts Focus After Phase II Failure In PAH” – Scrip, 16 Jan, 2018.)

HGPS is a rare genetic disorder with symptoms that resemble aspects of aging but at an early age. Though there is a heritable form, progeria usually occurs as a new point mutation in the LMNA gene, with a frequency of one per 8 million live births.

Eiger began a rolling NDA for Zokinvy, a farnesyl transferase inhibitor (FTI), last December, which it completed in March; the FDA has given a 20 November PDUFA date. The product also has breakthrough therapy designation for the treatment of HGPS.

Results from the ProLon1 and ProLon2 trials, which evaluated Zokinvy’s impact on mortality in progeria patients compared with non-treatment in contemporaneous untreated patients, were published in the Journal of the American Medical Association in April 2018. The combined data from the two studies showed that treatment with Zokinvy was associated with a lower mortality rate (HR 0.23; 95% CI, 0.06-0.90; p=0.04) after 2.2 years of follow-up.

Zokinvy is also in Phase III trials for the treatment of hepatitis D, with promising results from a Phase II trial released in October 2019. “Having one FDA approval under its belt would bode well for Zokinvy when Eiger eventually pursues approval of the drug for HDV. In addition, as the only approved product in the US for HGPS, Zokinvy would strengthen the company’s revenue stream to help fund future clinical trials and regulatory submissions for HDV,” said the Biomedtracker analysts.

Alnylam’s Lumasiran
Indication: Hyperoxaluria

PDUFA action date: 3 December NDA – First Review

If approved, Alnylam Pharmaceuticals Inc.’s lumasiran will be the first marketed therapy for the ultra-rare disease primary hyperoxaluria type 1 (PH1), and the third RNAi therapy to be approved, following the company’s pioneering success with patisiran in 2018.

The siRNA therapeutic targets hydroxyacid oxidase 1 (HAO1), which encodes glycolate oxidase. It has been granted breakthrough designation and is currently under priority review as a treatment for PH1, an ultra-rare disease that is estimated to have a prevalence of approximately 3,000–5,000 patients across the US and the EU. The disease is characterized by overproduction of liver oxalate, causing accumulation of oxalate in the kidneys, with progressive decline in kidney function, typically culminating in kidney failure.

Results from the Phase III ILLUMINATE-A study showed that at six months, lumasiran lowered patients’ urinary oxalate levels by 65% compared with baseline and by 54% compared with placebo. In addition, 52% of lumasiran-treated patients achieved urinary oxalate levels within normal range and 84% achieved near normal levels, whereas none of the patients on placebo achieved normal or near-normal levels.

Although a clinical benefit was not seen, the trial may have been … Next Page »

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