Rare dis­eases, by de­f­i­n­i­tion, are small cat­e­gories with few­er pa­tients. Yet, the in­di­vid­u­als and fam­i­lies deal­ing with some of the 7,000+ rec­og­nized rare con­di­tions share com­mon ex­pe­ri­ences. De­layed di­ag­noses, in­sur­ance chal­lenges and find­ing physi­cian ex­per­tise are typ­i­cal chal­lenges across most rare con­di­tions.

So Health Union is cre­at­ing a new on­line fo­rum to con­nect peo­ple un­der a broad­er rare dis­ease um­brel­la — and al­so give phar­ma com­pa­nies an­oth­er way to con­nect with those pa­tient in­flu­encers. Health Union’s es­tab­lished so­cial net­work of pa­tient groups in­cludes eight spe­cif­ic rare dis­ease groups, along with dozens of more com­mon health con­di­tions from asth­ma to thy­roid eye dis­ease, but the new RareDis­ease.net aims to in­clude any­one with a rare con­di­tion.

The phar­ma con­nec­tions through Health Union can help those look­ing for rare dis­ease pa­tient lead­ers for re­search, in­sights or mar­ket­ing ef­forts as in­flu­encer spokes­peo­ple. Health Union de­buted its So­cial Health Net­work prac­tice in June that not on­ly con­nects pa­tients to each oth­er, but al­so of­fers ad­vo­ca­cy skill train­ing and paid health­care com­pa­ny op­por­tu­ni­ties.

Am­ri­ta Bhowmick

“It’s a win-win be­cause phar­ma, of course, is try­ing to reach these pa­tients, but pa­tients are al­so try­ing to learn more about treat­ment op­tions, clin­i­cal tri­als, di­ag­nos­tics, etc.,” said Am­ri­ta Bhowmick, Health Union’s chief com­mu­ni­ty of­fi­cer. “And it’s even more im­por­tant for this com­mu­ni­ty be­cause it’s so un­der­served and it’s hard­er for peo­ple to get in­for­ma­tion.”

At RareDis­ease.net, new­ly di­ag­nosed pa­tients and fam­i­lies can browse con­tent about rare dis­ease ba­sics, di­ag­noses, man­age­ment, clin­i­cal tri­als, ad­vo­ca­cy and more. Its so­cial me­dia ac­counts on Face­book, Twit­ter and In­sta­gram will serve as gath­er­ing and con­tent-shar­ing hubs. Health Union is al­so plan­ning fu­ture spe­cial live events that will criss-cross rare dis­eases.

Col­lec­tive­ly, an es­ti­mat­ed one in 10 peo­ple in the US (about 30 mil­lion) have a rare dis­ease, and on­line health com­mu­ni­ties have proven to be im­por­tant, Bhowmick said.

“There’s been mas­sive growth, aware­ness and in­ter­est — and need — around rare dis­eases and a lot of that is the pow­er of the in­ter­net bring­ing peo­ple to­geth­er and dri­ving aware­ness, but it’s al­so in­creas­ing di­ag­no­sis rates and iden­ti­fi­ca­tion of these dis­eases,” she said. “…What’s in­ter­est­ing is there’s a bit of a Venn di­a­gram over­lap with many peo­ple with vary­ing rare con­di­tions hav­ing sim­i­lar ex­pe­ri­ences and chal­lenges.”

The FDA has rejected Incyte’s extended-release formulation of ruxolitinib tablets, in a surprise setback for the company’s plans to build on its blockbuster Jakafi franchise.

The ruxolitinib XR tablets are designed to be taken once a day, whereas Jakafi is indicated for twice daily dosage (although some patients can take it once daily).

According to Incyte, the FDA acknowledged in its complete response letter that the study submitted in the NDA “met its objective of bioequivalence based on area under the curve (AUC) parameters but identified additional requirements for approval.”

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”

A small, Santa Ana-based biotech created in 2017 is looking to enter a SPAC deal as it lays out plans to begin trials in its lead cell therapy candidates and bring on new executives.

Graf Acquisition Corp. IV and NKGen Biotech announced Thursday, with few other details, that the two companies signed a non-binding letter of intent to “pursue a business combination.” Graf Acquisition II and III withdrew their IPOs last year.

Cyclerion Therapeutics may have the design of a Phase IIb study ready to go, but it’s scrambling for a way to fund it.

The company said in a press release that it’s “actively evaluating the best combination of capital, capabilities, and transactions available to it to advance the development of zagociguat,” its lead candidate for a rare, genetic mitochondrial disease known as MELAS.

In a separate SEC filing, Cyclerion once again flagged “substantial doubt about (its) ability to continue as a going concern.” As of the end of 2022, it had cash and cash equivalents of only $13.4 million.