Go or no go? New Year approval decisions for the US regulators

Aurinia’s only project is due in front of the FDA, and outcomes are expected for Merck KGaA, Bayer and Merck & Co.

Company events

As 2020 draws to a close the US FDA has, to date, dished out 53 novel drug approvals. That number, which excludes diagnostics, blood reagents and emergency use authorisations, beats last year’s tally of 49, but manufacturing inspection delays due to the coronavirus pandemic have led to rejections and Pdufa extensions, a trend that will likely continue into next year.

Aurinia is expecting a decision on its sole project, the calcineurin inhibitor voclosporin. However, just last week Glaxosmithkline’s Benlysta received a thumbs up from the US regulator in lupus nephritis, the indication Aurinia is targeting.

Lupus nephritis is a serious complication of systemic lupus erythematosus (SLE) that affects the kidneys; approximately 50% of patients with SLE will develop nephritis.

In voclosporin’s Aurora study the oral project showed an 18.3-point benefit over the control arm on the primary endpoint of renal response at one year.

Glaxo’s Benlysta was first approved in the US for SLE in 2011, and can now add nephritis to its label on the back of Bliss-LN data. In that study intravenous Benlysta showed an 11-point benefit over control on the primary measure of renal response at two years. Both intravenous and subcutaneous versions of Benlysta are approved for nephritis.

Other big pharma competitors are on the horizon: Astra’s anifrolumab is filed in SLE, and the phase II Tulip-LN1 study in nephritis is due to yield data in the second quarter. Also, the phase III Regency study of Roche’s Gazyva in nephritis started earlier this year.

Aurinia wants to launch voclosporin by itself in the US. The company last week signed a deal with Otsuka covering Europe and Japan, but some investors were hoping for an outright acquisition, and shares fell 15% on the news.

A tale of two Mercks

Merck & Co and Bayer’s heart failure pill vericiguat looks set to arrive in a competitive market. The guanylate cyclase agonist is filed in patients with symptomatic chronic heart failure with reduced ejection fraction (HFrEF), a market where Novartis’s Entresto is becoming increasingly entrenched, and where Astrazeneca’s Farxiga gained approval earlier this year.

In the Victoria study, vericiguat failed to have an impact on cardiovascular death, with the win on the primary composite endpoint driven by hospitalisation numbers. However, the drug might be able to find a niche: the Victoria study was the first to show a benefit in a specific group of very sick patients.

Still, 2026 sellside consensus for vericiguat is modest, at $389m, versus the $4bn Entresto is forecast to bring in that year, according to EvaluatePharma data.

Elsewhere, Merck KGaA’s targeted lung cancer therapy tepotinib will be up before the regulators in the first quarter, but its label could come with a safety warning. The cMet inhibitor, approved as Tepmetko in Japan, has been filed in the US for metastatic NSCLC driven by the exon 14 skipping mutation.

The filing was based on the single-arm phase II Vision study, which showed an overall response rate of 59% when tepotinib was used as a first-line treatment, and 45% ORR in second/third-line use. Peripheral oedema of any grade was the most common treatment-related adverse event (63%), and there was one death, from respiratory failure and dyspnea secondary to interstitial lung disease, which was considered related to treatment.

Incyte and Novartis’s competing project Tabrecta was given an accelerated FDA greenlight in May and its own label includes a precaution about interstitial lung disease/pneumonitis, which led to a patient death in the phase II Geometry mono-1 study.

The tables below list first-time and supplementary US approvals due next month, with consensus forecasts from EvaluatePharma.

Notable first-time US approval decisions due in January
Project Company PDUFA date 2026e sales by indication ($m)
Ansofaxine hydrochloride Luye Pharma Jan 8 (estimate) -
Vericiguat Merck & Co/Bayer Jan 20 389
Voclosporin Aurinia Jan 22 1,114
Merck KGaA Q1 489
Sources: EvaluatePharma & company releases.


Supplementary and other notable approval decisions in January
Product Company Indication (clinical trial) Date
Xalkori Pfizer Paediatric patients relapsed or refractory systemic ALK-positive anaplastic large cell lymphoma (study ADVL0912 and A8081013) Jan
Nplate Amgen Hematopoietic syndrome of acute radiation syndrome Jan 28
Enhertu Astrazeneca Her2 +ve metastatic gastric or gastroesophageal junction adenocarcinoma (Destiny-Gastric01) Q1
Xolair Roche Self-administration option across all approved US indications Q1
Botox Abbvie Detrusor (bladder muscle) overactivity, paediatric patients Q1
Estelle Mithra To prevent pregnancy Estimated H1
Sources: EvaluatePharma & company releases.

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