With further FDA guidance yet to come on how fecal microbiota transplants (FMTs) to treat Clostridium difficile (C. diff) infections will be regulated, investors continue to weigh the odds of drugs vs. the controversial but popular therapy.
Trial designer and steering committee member Bruce Cree told BioWorld that the FDA made "a very wise decision" in assigning the broad label to Novartis AG's oral sphingosine 1-phosphate (S1P) receptor modulator Mayzent (siponimod) in multiple sclerosis (MS), "covering the continuum from clinically isolated syndrome all the way into active secondary progressive MS [SPMS]."
On the PDUFA date for Zynquista (sotagliflozin), the FDA delivered a complete response letter (CRL) to Lexicon Pharmaceuticals Inc. and partner Sanofi SA related to the NDA for the type 1 diabetes therapy, an oral dual inhibitor of sodium glucose transporter (SGLT) 1 and 2 that was previously known as LX-4211. Recro Pharma Inc. also found a CRL in its mailbox, related to the NDA for intravenous (I.V.) meloxicam for the management of moderate to severe pain.
Now that Dublin-based Jazz Pharmaceuticals plc has gained FDA marketing clearance for Sunosi (solriamfetol), investors are waiting to see how the DEA will schedule the compound, and J.P. Morgan analyst Jessica Fye said her "base case for DEA scheduling for Sunosi is [schedule] IV." The human abuse liability study compared the drug to phentermine and Sunosi "appeared less 'likable' than that schedule IV agent (note that [methylphenidate and d-amphetamine] are schedule II given their higher potential for abuse/dependence)," Fye wrote in a report.
The widely expected approval of Sage Therapeutics Inc.'s 60-hour intravenous (I.V.) Zulresso (brexanolone) in postpartum depression (PPD) opens the marketing door to the first therapy approved in the indication and raised investor hopes for Marinus Therapeutics Inc., developing a similar PPD therapy in I.V.-to-oral form as well as an all-oral version.
Urovant Sciences Ltd.'s favorable top-line data from the phase III study called Empowur with vibegron in adults with overactive bladder (OAB) did not bring the stock-boosting effect that some might have expected, as doubters fretted that the once-daily beta-3 adrenergic receptor agonist did not perform well enough relative to generic tolterodine, even though – as J.P. Morgan analyst Eric Joseph pointed out – the latter was not meant for a head-to-head comparison.
Aerpio Pharmaceuticals Inc.'s phase IIb data with AKB-9778 in moderate to severe nonproliferative diabetic retinopathy (NPDR) failed to provide betterment relative to the phase IIa experiment in diabetic macular edema (DME), but CEO Stephen Hoffman noted that the findings were "hot off the press" and the company needs to conduct an internal review, with more detail to come at a scientific meeting.
The prospects of an oral taxane chemotherapy – providing similar efficacy to intravenous (I.V.) with a better toxicity profile – have drawn players to the field that include Odonate Therapeutics Inc. and Athenex Inc., the latter with a phase III trial fully enrolled and the former with a late-stage experiment expected to finish sign-ups in the second half of this year.
As the Philippine Department of Justice (PDOJ) said it found probable cause to indict Sanofi SA and government officials over deaths caused by the Paris-based pharma giant's dengue vaccine, the FDA's Vaccines and Related Biological Products Advisory Committee (VRBPAC) reviewed the BLA for the product in an uneventful session.
Zafgen Inc. CEO Jeffrey Hatfield said the firm has backup compounds for ZGN-1258 to treat Prader-Willi syndrome (PWS) and "remains committed" to the condition. "We're going to evaluate all of our options to try and unlock potential value we see in methionine aminopeptidase 2 [MetAP2] inhibition for this terrible disease."
