When Pro­tal­ix Bio­Ther­a­peu­tics re­port­ed its 2018 re­sults this March, it spent much of its time talk­ing up the po­ten­tial of nab­bing an ac­cel­er­at­ed ap­proval for its Fab­ry dis­ease treat­ment pe­gu­ni­gal­si­dase al­fa.

Weeks lat­er, the Is­raeli biotech — which has an un­ap­pe­tiz­ing pen­ny stock val­u­a­tion $PLX — and part­ner Chiesi are go­ing for it, op­ti­mistic af­ter months of dis­cus­sion with the FDA. The BLA fil­ing is sched­uled for Q1 2020.

Like Shire’s Re­pla­gal, which it’s be­ing com­pared against in a pair of late-stage tri­als, pe­gu­ni­gal­si­dase al­fa (or PRX-102) is an en­zyme re­place­ment ther­a­py de­signed to break down the ex­cess fat mol­e­cules build­ing up in Fab­ry pa­tients. Syn­the­sized from plants, this par­tic­u­lar re­com­bi­nant al­pha-galac­tosi­dase A en­zyme can be in­fused every four weeks in­stead of Re­pla­gal’s two.

An ini­tial glimpse of the Phase III study showed that high lev­els of the drug cir­cu­lat­ed in the blood­stream af­ter 28 days, with a mean con­cen­tra­tion of 138 ng/mL among 15 evalu­able pa­tients. In con­trast, Fab­razyme — an­oth­er ERT de­vel­oped by Sanofi Gen­zyme — had his­tor­i­cal­ly shown a mean con­cen­tra­tion of 20 ng/mL at 10 hours post in­fu­sion, HC Wain­wright an­a­lysts added in a Feb­ru­ary note.

But those are not the on­ly com­peti­tors Pro­tal­ix will face. Am­i­cus has trav­eled down its own wind­ing path to an ap­proval for Galafold, an oral drug that works by ad­dress­ing some mis­fold­ed α-GalA en­zymes. New ther­a­pies to in­tro­duce healthy copies of the GLA gene, which en­codes for the en­zyme, are com­ing up in the pipeline, with a no­table ef­fort from Avro­bio.

“This reg­u­la­to­ry ap­proval path is a sig­nif­i­cant achieve­ment as it means that we can start the ap­pli­ca­tion process and po­ten­tial­ly at­tain mar­ket ap­proval sig­nif­i­cant­ly ear­li­er than the ini­tial plan of da­ta from our on­go­ing phase III BAL­ANCE clin­i­cal tri­al,” CEO Moshe Manor said in a state­ment. “We plan to con­tin­ue the BAL­ANCE study to fur­ther strength­en the pro­file of pe­gu­ni­gal­si­dase al­fa.”

Chiesi holds the US rights to the drug from a deal that looped in $760 mil­lion in reg­u­la­to­ry and com­mer­cial mile­stones. Pro­tal­ix cur­rent­ly has one ap­proved prod­uct for Gauch­er dis­ease, mar­ket­ed by Pfiz­er.

That 2012 ap­proval, though, has done lit­tle to lift Pro­tal­ix’s suf­fer­ing stock af­ter a con­found­ing de­ci­sion in 2007 to price a sec­ondary of­fer­ing at an 85% dis­count to its trad­ing price the day pri­or. Shares plum­met­ed from $35 to $5 overnight and has bare­ly gone over $10 since, trad­ing way be­low $1 these days.

So­cial im­age: Shut­ter­stock

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Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.