Or­p­hazyme’s bet that, de­spite miss­ing both pri­ma­ry end­points on its piv­otal study, they would find wel­com­ing ears at the FDA has paid off, for now.

The Dan­ish biotech an­nounced to­day that the FDA has ac­cept­ed the ap­pli­ca­tion for their ex­per­i­men­tal drug ari­mo­clo­mol in the rare neu­rode­gen­er­a­tive con­di­tion Nie­mann-Pick dis­ease Type C and giv­en it pri­or­i­ty re­view. Al­though drugs that fail piv­otal stud­ies rarely re­ceive a pri­or­i­ty look at the agency, the dis­tinc­tion should come with lit­tle sur­prise. Or­p­hazyme has sig­naled re­peat­ed­ly since the 2019 re­sults came out that the FDA viewed an ap­pli­ca­tion fa­vor­ably and, the FDA gave it break­through des­ig­na­tion that No­vem­ber.

The agency set a PDU­FA date for March 17, 2021. Or­p­hazyme’s stock jumped 8.38% on the news, from 83.5 kro­nes to 90.5 kro­nes ($13.3 to $14.3).

“We look for­ward to col­lab­o­rat­ing with the FDA as they com­plete their re­view of this NDA to ad­dress the un­met med­ical need in NPC, and mean­while are work­ing to ex­pand our U.S. ac­tiv­i­ties in prepa­ra­tion for po­ten­tial com­mer­cial avail­abil­i­ty next year,” CEO Kim Strat­ton said in a state­ment.

Or­p­hazyme, a one-drug com­pa­ny tar­get­ing a string of neu­rode­gen­er­a­tive dis­or­ders, came out it with re­sults from a 50-per­son Phase II/III study on Nie­mann-Pick type C pa­tients, their lead in­di­ca­tion, in Jan­u­ary 2019. Ari­mo­clo­mol failed to hit both pri­ma­ry end­points, both a stan­dard scale for as­sess­ing symp­toms in pa­tients, called the NPC Clin­i­cal Sever­i­ty Scale, and the Clin­i­cal Glob­al Im­pres­sion of Im­prove­ment, a self-rat­ing tool that the FDA re­quest­ed. It al­so failed sev­er­al sec­ondary end­points.

Still, the drug on­ly nar­row­ly missed sig­nif­i­cance on the first pri­ma­ry end­point, where they saw both a 74% re­duc­tion in dis­ease sever­i­ty and a p-val­ue of 0.0506. And the com­pa­ny not­ed that two pre-spec­i­fied sub­group analy­ses reached sig­nif­i­cance, with dis­ease pro­gres­sion slow­ing in pa­tients over the age of 4 and in pa­tients who had been tak­ing an Acte­lion drug some­times pre­scribed for the dis­ease. All end­points were against place­bo.

In a de­gen­er­a­tive con­di­tion with no sig­nif­i­cant treat­ments, Or­p­hazyme was will­ing to bet that was enough to take to reg­u­la­tors. So far, they’ve been proven right.

Or­p­hazyme, eye­ing funds for com­mer­cial­iza­tion, filed for its first US of­fer­ing ear­li­er this month, pen­cil­ing in an ini­tial fig­ure of $115 mil­lion for the raise. In ad­di­tion to their lead in­di­ca­tion, they al­so have piv­otal stud­ies on­go­ing for ALS and spo­radic in­clu­sion body myosi­tis, a pro­gres­sive mus­cle dis­or­der.

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.