Five years af­ter EU reg­u­la­tors ap­proved a ther­a­py to treat a rare in­her­it­ed meta­bol­ic con­di­tion that caus­es ex­treme sen­si­tiv­i­ty to light, the FDA has en­dorsed the treat­ment — mark­ing the first-ever ap­proval for pa­tients with ery­thro­poi­et­ic pro­to­por­phyr­ia (EPP) in the Unit­ed States.

The ther­a­py, which is ad­min­is­tered via an im­plant in­sert­ed sub­cu­ta­neous­ly, was de­vel­oped by Aus­tralian drug mak­er Clin­u­v­el Phar­ma­ceu­ti­cals and is sold as Sce­nesse.

In pa­tients with EPP, the ac­tiv­i­ty of fer­rochelatase — an en­zyme in­volved in the pro­duc­tion of a key com­po­nent in he­mo­glo­bin — is com­pro­mised. Re­duced fer­rochelatase ac­tiv­i­ty caus­es the ac­cu­mu­la­tion of pro­to­por­phyrin IX (PPIX) — and light ex­po­sure on the skin can re­act with PPIX, spark­ing in­tense pain, red­ness and thick­en­ing.

Sce­nesse, known chem­i­cal­ly as afame­lan­otide, binds with the melanocortin-1 re­cep­tor on the sur­face of melanocyte cells found in the epi­der­mis of the skin. This trig­gers a cas­cade of re­ac­tions that re­sult in the melanocyte fa­vor­ing the pro­duc­tion of eu­me­lanin (black/brown) over pheome­lanin (red/yel­low). The pro­duc­tion of eu­me­lanin in the skin in­de­pen­dent of ex­po­sure to sun­light or ar­ti­fi­cial light sources of­fers the pa­tient the abil­i­ty to cope with the mol­e­c­u­lar dam­age caused by light.

In Jan­u­ary, STAT pro­filed a Con­necti­cut woman who com­pared the pain she felt from light ex­po­sure to the ex­pe­ri­ence of hav­ing boil­ing oil poured on her hands and knees. On­ly around 600 Amer­i­cans suf­fer from EPP. Switzer­land and Italy had Clin­u­v­el agree to pay 80% of the drug’s cost be­fore al­low­ing it, ac­cord­ing to STAT.

“We are de­ter­min­ing our pric­ing strat­e­gy (in the Unit­ed States) but our ap­proach is go­ing to be far more trans­par­ent and fair than many of our peers. For this we re­fer you to our ap­proach to pric­ing in Eu­rope,” a Clin­u­v­el spokesper­son told End­points News.

Sce­nesse is priced in Eu­rope at €14,101 per in­jectable im­plant, or €56,404 per pa­tient based on four in­jectable im­plants per an­num (ex­clud­ing lo­cal tax­es), the spokesper­son added.

The US ap­proval for Sce­nesse was based on twin place­bo-con­trolled clin­i­cal tri­als. In the first 93-pa­tient tri­al, 48 sub­jects were giv­en Sce­nesse, while the rest were on place­bo.  Af­ter 180 days of ex­po­sure to di­rect sun­light be­tween 10 am and 6 pm, pa­tients on the ther­a­py were pain-free for a me­di­an 64 hours, ver­sus 41 hours for those in the place­bo arm.

In the oth­er 74-pa­tient study, 38 re­ceived Sce­nesse, while the rest got place­bo. Pa­tients were fol­lowed for 270 days which they spent out­doors be­tween 10 am and 3 pm and were as­sessed for pain lev­els — al­though the analy­sis did not in­clude sun ex­po­sure on days pa­tients re­port­ed spend­ing time in a com­bi­na­tion of both di­rect sun­light and shade. The me­di­an to­tal num­ber of hours un­der these con­di­tions in which pa­tients felt ‘no pain’ was six hours for pa­tients re­ceiv­ing Sce­nesse and 0.75 hours for pa­tients re­ceiv­ing place­bo.

Clin­u­v­el formed in 1987 af­ter re­searchers start­ed work­ing on the idea of syn­the­siz­ing hu­man hor­mones to pro­tect the skin.

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).

It is no secret that drug shortages have been prevalent in 2022. Several major drug products, such as amoxicillin and Adderall, have been in short supply for several months and have led to members of Congress applying pressure on the FDA and HHS to resolve the situation.

Speaking at a webinar hosted by the Alliance for a Stronger FDA, Valerie Jensen, the associate director of the FDA’s Drug Shortage Staff, noted both the rise in quality-related issues and increased demand for some products. She called on companies to report such demand increases, even though they are not currently required to do so.