Re­gen­eron has held high hopes for evinacum­ab, a rare cho­les­terol dis­or­der drug that earned a first-in-class nod from the FDA ear­ly last year, as a cen­ter­piece of its car­diometa­bol­ic fran­chise. Now, the com­pa­ny is tap­ping an ul­tra-rare spe­cial­ist to take evinacum­ab to the next lev­el abroad.

Re­gen­eron will re­ceive $30 mil­lion in up­front cash and a po­ten­tial $63 mil­lion in down­stream mile­stones from Ul­tragenyx for ex-US li­cens­ing rights to Evkeeza (evinacum­ab), an ANGPTL3 block­er with an FDA ap­proval to treat a rare cho­les­terol dis­or­der along­side LDL-C low­er­ing ther­a­py and di­et, the com­pa­nies said Fri­day.

Ul­tragenyx, a small phar­ma known best for its ul­tra-rare dis­ease drugs, will pick up de­vel­op­ment and com­mer­cial­iza­tion costs for evinacum­ab in pa­tients with the con­di­tion known as ho­mozy­gous fa­mil­ial hy­per­c­ho­les­terolemia (HoFH), which is be­lieved to af­fect any­where be­tween 1 in 160,000 and 300,000 peo­ple world­wide.

In ad­di­tion, Re­gen­eron and Ul­tragenyx have set pre­lim­i­nary terms for an ex-US li­cens­ing deal for one of Re­gen­eron’s in­ves­ti­ga­tion­al drugs, an an­ti­body de­signed to treat the ul­tra-rare dis­ease fi­brodys­pla­sia os­si­f­i­cans pro­gres­si­va (FOP). Terms for that po­ten­tial part­ner­ship have yet to be set.

Evinacum­ab was ap­proved last Feb­ru­ary at the hefty price tag of an av­er­age of $450,000 per year. It was the first drug ap­proved to in­hib­it ANGPTL3, or an­giopoi­etin-like 3, a pro­tein in­volved in lipid me­tab­o­lism.

In piv­otal da­ta back­ing the ap­proval, pa­tients tak­ing evinacum­ab saw a 47% re­duc­tion in LDL lev­els from base­line, com­pared to a 2% in­crease in the con­trol. The drug arm al­so saw sig­nif­i­cant re­duc­tions in lev­els of apolipopro­tein B, an in­di­ca­tor of vas­cu­lar dis­ease, as well as triglyc­erides and over­all cho­les­terol.

Evinacum­ab was en­vi­sioned as a sort of nat­ur­al fol­low-up for Re­gen­eron’s Pralu­ent, a part­nered PC­SK9 block­er with Sanofi ap­proved to low­er LDL-C. That drug has been locked in a years­long pric­ing bat­tle with Am­gen’s Repatha in what has ef­fec­tive­ly be­come a war of at­tri­tion over price de­creas­es in the pricey PC­SK9 class.

Ul­tragenyx, mean­while, scored its first FDA ap­proval some five years ago with Mep­se­vii, a drug ap­proved to treat an ul­tra-rare ge­net­ic en­zyme dis­or­der called MPS VII. Since then, the drug­mak­er has earned its stature as an ul­tra-rare spe­cial­ist, bring­ing two ad­di­tion­al drugs on­to the mar­ket in Crysvi­ta, ap­proved to treat two forms of hy­pophos­phatemia, and Do­jolvi, an en­gi­neered medi­um-chain fat­ty acid de­signed as a sup­ple­ment for pa­tients with mol­e­c­u­lar­ly con­firmed long-chain fat­ty acid ox­i­da­tion dis­or­ders.

At the end of January, the European Medicines Agency officially launched its new clinical trials info system (CTIS), although the migration to the new platform has only really just begun, and sponsors have until the end of January 2023 before all initial trial applications must be submitted through CTIS.

Overall, 56 clinical trial applications have been submitted in CTIS during the first 3 months since the launch of the system on Jan. 31, according to new data posted by the EMA. By comparison, about 4,000 new trials are authorized each year across Europe.

As concerns related to uptake and distribution continue to linger, Switzerland is among the first countries that plans to destroy hundreds of thousands of expired and unused Covid-19 vaccine doses.

The European country said it plans to destroy more than 600,000 doses of Moderna’s Spikevax Covid-19 vaccine as the doses have reached their expiration date.

However, Moderna CEO Stéphane Bancel told the World Economic Forum in Davos, Switzerland that he’s in the process of throwing 30 million doses in the garbage, exclaiming, “We have a big demand problem.”

More than 500 stakeholders sent comments to the FTC on whether the commission should look further into pharma middlemen, known as PBMs, with many of the commenters calling for more federal oversight.

Similar to the critical open comment period in a deadlocked FTC session last February, pharmacies and pharmacy groups are continuing to call out the lack of transparency among the top 3 PBMs, which control about 80% of the market.

HHS on Thursday finalized its decision to withdraw a rule, proposed just before former President Donald Trump left office, that would’ve caused thousands of HHS and FDA regulations to automatically expire if they weren’t reviewed within two years, and every 10 years thereafter.

The decision follows the filing of a lawsuit last March, in which several nonprofits alleged that the outgoing administration planted “a ticking timebomb” for HHS, essentially forcing it to devote an enormous amount of resources to the unprecedented and infeasible task of reviewing thousands of regulations regularly.