The regulatory round-up: Eight key FDA decisions


The Food and Drug Administration (FDA) in the US has made several key drug decisions over the last few weeks. Here’s a summary.

Orserdu (elacestrant), Stemline Therapeutics

The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer was approved in the US in February.

The FDA approved Orserdu (elacestrant) for the treatment of postmenopausal women or adult men, with ER+, HER2-, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy.

Read more: FDA-approved therapy targets ESR1 mutations in breast cancer

Jesduvroq (daprodustat), GSK

The FDA approved Jesduvroq (daprodustat) for the once-a-day treatment of anaemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least four months.

This made Jesduvroq is the first oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) to be approved in the US.

Read more: New oral option for anaemia of CKD approved in US

EA-2353, Endogena Therapeutics

The investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) was designated as a Fast Track development programme in February.

EA-2353 takes a novel, small-molecule approach and selectively activates endogenous retinal stem and progenitor cells, which differentiate into photoreceptors and can potentially preserve or restore visual function. The drug was granted orphan drug designation by the FDA in May 2021.

Read more: FDA Fast Track Designation for retinitis pigmentosa treatment

Drug candidate for TUBB4a leukodystrophy, SynaptixBio

The FDA has granted Rare Paediatric Disease (RPD) Designation to SynaptixBio’s drug candidate for TUBB4a leukodystrophy – a genetic and debilitating condition which mainly affects babies and young children.

The designation is the first step to gaining a priority review voucher (PRV), which can accelerate market access for therapeutics. They can also be sold or traded by sponsors, such as big pharma corporations.

Read more: Treatment for leukodystrophy could be “revolutionary”

Jemperli (dostarlimab-gxly), GSK

The FDA granted full approval for Jemperli (dostarlimab-gxly) for the treatment of adult patients with mismatch repair-deficient (dMMR) recurrent or advanced endometrial cancer, as determined by a US FDA-approved test.

The approval is for endometrial cancer that has progressed on or following a prior platinum-containing regimen in any setting and are not candidates for curative surgery or radiation.

Read more: Full FDA approval for endometrial cancer treatment

Trodelvy (sacituzumab govitecan-hziy), Gilead Sciences

The FDA granted expanded approval for Trodelvy (sacituzumab govitecan-hziy) for patients with HR-positive/HER2-negative metastatic breast cancer who have received hormonal therapy and at least two additional therapies in the metastatic setting.

The drug is expected to become a standard of care for these patients, who have had limited treatment options, according to analysts GlobalData.

Read more: ADC potential “standard of care” for some breast cancer patients

NTLA-2002, Intellia Therapeutics

The FDA cleared Intellia Therapeutics’ Investigational New Drug (IND) application for NTLA-2002 for the treatment of hereditary angioedema (HAE) in March.

The decision will enable the company to include the US in the global Phase II portion of its ongoing Phase I/II study of NTLA-2002, an in vivo genome editing candidate designed to inactivate the target gene, kallikrein B1 (KLKB1).

Read more: FDA clears in vivo gene editing therapy for trials in US

RRx-001, EpicentRx

Most recently, the FDA granted Fast Track Designation to RRx-001 for the prevention/attenuation of severe oral mucositis in chemotherapy and radiation-treated head and neck cancer patients.

The regulator also accepted the company’s Investigational New Drug (IND) application to initiate a follow-on Phase IIb clinical trial, called KEVLARx, in the same head and neck cancer patient population.

Read more: US FDA fast-tracks drug to prevent severe chemotherapy side effect

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