FDA ap­proves drug for rare con­di­tion that trig­gers in­tense pain from light ex­po­sure

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.

Op­ti­miz­ing Cell and Gene Ther­a­py De­vel­op­ment and Pro­duc­tion: How Tech­nol­o­gy Providers Like Corn­ing Life Sci­ences are Spurring In­no­va­tion

Remarkable advances in cell and gene therapy over the last decade offer unprecedented therapeutic promise and bring new hope for many patients facing diseases once thought incurable. However, for cell and gene therapies to reach their full potential, researchers, manufacturers, life science companies, and academics will need to work together to solve the significant challenges facing the industry.

Pfiz­er, Sarep­ta and two oth­ers sug­gest Duchenne drug safe­ty is­sues tied to "class ef­fect"

Since the first experimental Duchenne gene therapy programs came about, the space has proven rife with safety issues and patient deaths in clinical trials. Pfizer and three biotechs now think they’ve found a reason why.

The four companies suggested there may be a “class effect” causing the adverse events in Duchenne gene therapies, they wrote in a new study. They specifically highlighted how side effects in five patients across three trials, who all showed muscle weakness with cardiac involvement, were “strikingly similar.”

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Pre­sent­ing a live End­points News event: Man­ag­ing a biotech in tur­bu­lent times

Biotech is one of the smartest, best educated industries on the planet. PhDs abound. We’ve had a long enough track record to see a new generation of savvy, experienced execs coming together to run startups.

And in these times, they are being tested as never before.

Biotech is going through quite a rough patch right now. For 2 years, practically anyone with a decent resume and some half-baked ideas on biotech could start a company and get it funded. The pandemic made it easy in many ways to pull off an IPO, with traditional road shows shut down in exchange for a series of quick Zoom meetings. Generalist investors flocked as the numbers raised soared into the stratosphere.

Frank Pallone (D-NJ), House Energy and Commerce Committee chair (Kevin Dietsch/Pool via AP Images)

House com­mit­tee unan­i­mous­ly ad­vances FDA user fee leg­is­la­tion with ac­cel­er­at­ed ap­proval tweaks

The House Energy and Commerce Committee on Wednesday offered a rare show of bipartisan support for a bill that would provide the FDA with user fees for the next five years.

The committee voted 55-0 to advance the quinquennial user fee bill to the full House floor, which if approved, will allow the FDA to use biopharma funds to hire new reviewers, and hit new marks as outlined in the user fee deals that the FDA and biopharma companies forged over the past several years.

Pearl Huang, former Cygnal Therapeutics president and CEO

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Flagship has crafted a new startup out of pieces from a pair of fledglings in the VC’s nest. And a prominent Roche veteran who ran one of the biotechs won’t be making the next leg of the journey.

The new company is called Sonata Therapeutics, which is picking up the work that Inzen was doing related to the cellular microenvironment and combining with Flagship’s Cygnal Therapeutics, which came out of stealth more than 3 years ago and put Pearl Huang — the BeiGene founder and former Roche SVP — at the helm.

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Patty Murray (D-WA) (Graeme Sloan/Sipa USA)(Sipa via AP Images)

Sen­ate user fee reau­tho­riza­tion bill omits ac­cel­er­at­ed ap­proval re­forms, shows wide gaps with House ver­sion

The Senate health committee on Tuesday released its first version of the bill to reauthorize all the different FDA user fees. But unlike the House version, there are only a few controversial items in the Senate’s version, which does not address either accelerated approval reforms or clinical trial diversity (as the House did).

While it’s still relatively early in the process of finalizing this legislation (the ultimate statutory deadline is the end of September), the House and Senate, at least initially, appear to be starting off in different corners on what should be included.

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FDA lob­bies Con­gress over rare dis­ease court rul­ing with wide im­pli­ca­tions

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Peter Marks (Greg Nash/Pool via AP)

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A DC appeals court clerk and researchers from Harvard and the University of Calgary dug through all the patents and regulatory exclusivities granted to inhalers approved by the FDA between 1986 and 2020, finding that of the 62 inhalers approved, 53 (or 85%) were brand-name products, with a median of 16 years of protection from generic competition.