New drug approvals in 2019 - what’s left to come…

For our recent analysis of novel drugs approved by the FDA in the year to date read this.

Below we take a look at what other new products are likely to be green-lit by the US regulator before year-end.

Selinexor - PDUFA July 6

Filed by Karyopharm for multiple myeloma

The good news…

Key opinion leaders think Selinexor is a promising agent with a novel mechanism of action. More here

The bad news…

Selinexor’s PDUFA date has already been pushed out by three months. Approval could be delayed again, into next year, if the FDA requires more data (see ViewPoints: Karyopharm gets temporary selinexor reprieve, but stakes are raised)

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Entrectinib - PDUFA August 16

Filed by Roche for NTRK mutated solid tumours / ROS1 positive non-small cell lung cancer

The good news…

Recently approved in Japan for NTRK mutated tumours and branded as Rozlytrek. Provides further compelling evidence of the potential for next-generation targeted cancer therapies (see ASCO19: Roche's entrectinib shrinks tumours in children, adolescents with NTRK, ROS1 or ALK gene fusions)

The bad news…

Entrectinib faces competition from Bayer’s already-launched Vitrakvi. Adoption of NTRK testing is likely to be slow despite compelling efficacy, say oncologists.

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Golodirsen - PDUFA August 19

Filed by Sarepta Therapeutics for Duchenne muscular dystrophy

The good news…

Approval would extend Sarepta’s current leadership of the DMD market. Golodirsen is designed to treat the approximate 8% of patients who have genetic mutations subject to skipping exon 53 of the dystrophin gene. The company also looks well positioned to lead the push into DMD gene therapies.

The bad news…

DMD has become a furiously competitive development space, including the recent entry of Vertex (see ViewPoints: DMD becomes bell of the biotech ball). 

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Upadacitinib - PDUFA August 19

Filed by AbbVie for rheumatoid arthritis

The good news…

Upadacitinib could become the most widely prescribed JAK inhibitor, say experts. Newer JAKs are likely to gain more share than their older counterparts.

The bad news…

It will enter a highly competitive market dominated by well-established biologics. Key opinion leaders suggest vigilance is required toward the potential frequency of thromboembolic events with upadacitinib in a real world setting.

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Fedratinib - PDUFA September 3

Filed by Celgene for myelofibrosis

The good news…

Fedratinib could offer a therapeutic option for patients who are ineligible for or who have previously failed available treatment, including Incyte and Novartis’ Jakafi.

The bad news…

It could, nevertheless, struggle to justify the $1.1 billion Celgene paid upfront to acquire Impact Biomedicines last January; that said, the outlook for Celgene has changed considerably since then.

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Oral semaglutide - PDUFA September 20

Filed by Novo Nordisk for type 2 diabetes

The good news…

Oral semaglutide is a potential game changer that could accelerate earlier use of the GLP-1 agonist mechanism. It is likely to be the only oral GLP-1 available for some time.

The bad news…

Novo Nordisk could face some tricky decisions regarding its pricing strategy for oral semaglutide. KOLs argue that the need to fast before the medication is taken may put some patients off.

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Vascepa - PDUFA September 28

Filed by Amarin for residual cardiovascular risk in patients with statin-managed LDL cholesterol and persistent elevated triglycerides

The good news

Vascepa has been granted priority review and looks increasingly unlikely to face an advisory committee review. Supporting data from the REDUCE-IT study could be practice changing.

See Physician Views Results - Cardiologist confidence in Vascepa grows as Amarin readies regulatory filing and KOL Views Results: Vascepa outpaces questions about placebo to score big win in REDUCE-IT, says leading cardiologist 

The bad news

Some question marks remain over performance of the placebo arm in REDUCE-IT and whether this has overstated Vascepa’s efficacy. Why hasn’t a long rumoured buy out materialised?

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RTH258/brolucizumab - PDUFA October 15

Filed by Novartis for age-related macular degeneration (AMD)

The good news

RTH258 appears to offer better efficacy than Regeneron and Bayer’s Eylea, and with less frequent dosing.

The bad news

Not all patients treated with RTH258 can be maintained on once-every-12-week dosing. A less frequently dosed formulation of Eylea has been approved by the FDA. What role will pricing therefore play?

See Physician Views snap-poll results: Ophthalmologists impressed with brolucizumab, but less frequent dosing for Eylea will act as a deterrent 

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Darolutamide - PDUFA October 25

Filed by Bayer for prostate cancer

The good news

Darolutamide has been granted priority review status by the FDA for non-metastatic castration-resistant prostate cancer. Approval will provide much needed momentum for Bayer’s late stage pipeline.

The bad news

Key opinion leaders have questioned whether darolutamide can stand out from already approved prostate cancer therapies.

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Lasmiditan - PDUFA November 14

Filed by Eli Lilly for acute migraine

The good news

Lasmiditan is a potential first-in-class agent providing an alternative mechanism of action. Key opinion leaders are appreciative of data showing a clean cardiovascular safety profile.

The bad news

Their overall perception, however, is of efficacy that is comparable to established fast-acting triptans. In addition, the fact that lasmiditan can cross the blood-brain barrier potentially causing dizziness is a matter of significant KOL concern.

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Ubrogepant - PDUFA November

Filed by Allergan for acute migraine

The good news

Ubrogepant could become a first-in-class oral CGRP inhibitor.

The bad news

KOLs are largely unimpressed with ubrogepant’s efficacy, suggesting it will be hard to gain market share from well-established triptans without aggressive pricing.

Allergan is being acquired by AbbVie, which has ascribed little value to the former’s late stage pipeline.

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Luspatercept - PDUFA December 4

Filed by Celgene for myelodysplastic syndromes

The good news

Analysts at Wolfe Research recently described luspatercept as having “seemingly great data,” in an area of high unmet need.

The bad news

With completion of Bristol-Myers Squibb’s acquisition of Celgene pushed into the fourth quarter will luspatercept get the attention at launch it deserves?

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BIIB098 - PDUFA December 24

Filed by Biogen for multiple sclerosis

The good news

Experts suggest that a more tolerable version of Biogen’s Tecfidera (essentially what BIIB098 is) is a winning strategy on paper. Head to head data showing whether BIIB098 significantly improves gastrointestinal tolerability is due imminently.

The bad news

KOLs worry that BIIB098 may struggle to secure reimbursement and it remains unclear how much new market share (versus cannibalisation of Tecfidera) it may achieve.

To read more ViewPoints articles, click here.

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