Although August is a fairly busy month for PDUFA dates, there were only two on the U.S. FDA’s calendar for this week, and one of those has already been reported. Here’s a look.
Although August is a fairly busy month for PDUFA dates, there were only two on the U.S. Food and Drug Administration (FDA)’s calendar for this week, and one of those has already been reported. Here’s a look.
Medexus’ Treosulfan for Hematopoietic Stem Cell Transplant Prep
Medexus Pharmaceuticals, based in Toronto and Chicago, had a target action date of August 11 for its New Drug Application (NDA) for treosulfan in combination with fludarabine as preparation for allogeneic hematopoietic stem cell transplantation (allo-HSCT). On August 3, the company and its development partner, medac GmbH, announced they had received a Complete Response Letter (CRL) from the FDA.
A preparative regimen is given to the patient before stem cell transplantation with the goal of decreasing tumor burden. And in the case of an allogeneic transplantation, allows for engraftment of the donor cells.
The CRL indicated it couldn’t be approved in its present form. It offered specific recommendations, including additional clinical and statistical data related to the primary and secondary endpoints of the pivotal Phase III trial. The companies say they are reviewing the CRL to decide on a course of action.
Medexus, headquartered in Toronto, Ontario, Canada, and medac, based in Wedel, Germany, inked a licensing deal on July 12 to commercialize treosulfan in Canada. It is being marketed in Canada under the name Trecondyv. Medexus will handle sales and marketing, while medac will handle manufacturing and supply.
The therapy has been distributed in Canada under the Special Access Program and recently received approval for commercialization from Health Canada for adults with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS) who are at increased risk for standard conditioning therapies. It was also approved for children older than one year old with AML or MDS.
Ken d’Entremont, chief executive officer of Medexus, said, “Given the recent Health Canada approval, European Medicines Agency approval in 2019, as well as supporting data from more than 100 publications, we were all surprised by the FDA’s response. That being said, Medexus and medac look forward to continuing to work with the FDA to address their requests in a timely manner, and we remain optimistic for a future, albeit delayed, approval of treosulfan in the United States, complete with Orphan Drug Designation.”
Jazz Pharma’s Xywav for Idiopathic Hypersomnia
Dublin-based Jazz Pharmaceuticals has a target action date of August 12 for its supplemental New Drug Application (sNDA) for Xywav (calcium, magnesium, potassium, and sodium oxybates) oral solution in adults with idiopathic hypersomnia. Idiopathic hypersomnia is a neurologic sleep disorder marked by chronic excessive daytime sleepiness not caused by other medical, behavioral or psychiatric conditions. Xywav has been approved by the FDA for the treatment of cataplexy or excessive daytime sleepiness in patients seven years of age and older with narcolepsy.
The sNDA was based on results from the Phase III trial of the drug in a double-blind, multicenter, placebo-controlled, randomized withdrawal study. The trial hit the primary endpoint of clinically meaningful improvements in the Epworth Sleepiness Scale (ESS) and the key secondary endpoints of Patient Global Impression of change and Idiopathic Hypersomnia Severity Scale (IHSS).
On August 3, Jazz released its second-quarter financial report, with Xywav creating net product sales of $124.2 million for the quarter. In June 2021, the FDA granted Xywav seven years of Orphan Drug Exclusivity, and the agency also published a summary of clinical superiority for Xywav, stating that the drug is clinically superior to Xyrem because of greater safety — Xywav creates a significantly decreased chronic sodium burden compared to Xyrem, which is clinically meaningful.
Jazz’s chairman and chief executive officer, Bruce Cozadd, said, “We have now executed four of five planned product launches since the beginning of 2020 and look forward to our anticipated launch of Xywav in idiopathic hypersomnia later this year, a critical step forward for these underserved patients. With 41% of our second quarter net product sales from recently launched or acquired products, we are well on track to meet our revenue diversification targets while driving significant shareholder value.”
