Af­ter re­ject­ing it twice, the FDA has fi­nal­ly ap­proved a new drug for low­er­ing the risk of hear­ing loss among chil­dren be­ing treat­ed for can­cer with chemother­a­py.

Ped­mark, as Fen­nec Phar­ma calls its drug, is a for­mu­la­tion of sodi­um thio­sul­fate. Giv­en that oto­tox­i­c­i­ty is a ma­jor dose-lim­it­ing side ef­fect of cis­platin chemother­a­py and there were no avail­able treat­ments for it, the FDA grant­ed North Car­oli­na-based Fen­nec both fast track and break­through ther­a­py des­ig­na­tions back in 2018, and put the drug on pri­or­i­ty re­view two years lat­er.

But man­u­fac­tur­ing is­sues that turned up at a pre-ap­proval in­spec­tion and were doc­u­ment­ed in a Form 483 ul­ti­mate­ly led to a com­plete re­sponse let­ter from the FDA. A re­sub­mis­sion one year lat­er failed to change the FDA’s minds.

Then this April, Fen­nec filed for ap­proval a third time.

In its re­cent quar­ter­ly re­port, the com­pa­ny not­ed that it’s built out the com­mer­cial or­ga­ni­za­tion over the past months with key hires in sales, mar­ket ac­cess and field-based med­ical teams.

Ped­mark is in­di­cat­ed for pe­di­atric pa­tients be­tween 1 month to 18 years old who have lo­cal­ized, non-metasta­t­ic sol­id tu­mors and have been treat­ed with cis­platin in­fu­sions for no longer than six hours. For this group of pa­tients, hear­ing loss stem­ming from such chemother­a­pies is of­ten long-last­ing and per­ma­nent, and cur­rent­ly they would need to re­sort to cochlear im­plants.

Com­mon side ef­fects in­clude hy­per­sen­si­tiv­i­ty, hy­per­na­trem­ia (hav­ing too much sodi­um in the blood), hy­pokalemia (hav­ing too lit­tle potas­si­um), nau­sea and vom­it­ing.

Shares $FENC rose al­most 16% to $7.63.

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

A Cipla drug manufacturing site in India has once again landed in the crosshairs of FDA inspectors.

The facility in question is Cipla’s drug manufacturing facility in the village of Verna, in the state of Goa in India’s southwest. In a sign that foreign inspections might ramp up again, the FDA’s visit from Aug. 16 to Aug. 22 uncovered six observations.

The 11-page report noted that environmental monitoring at the site did not properly ensure that microbial contaminants were not making any impact in the aseptic filling areas. It also found that procedures meant to stop microbial contamination were not adequately conducted in aseptic areas of the facility.

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The FDA’s Oncologic Drugs Advisory Committee on Friday voted 8-4 against the benefit-risk profile of Secura Bio’s PI3K inhibitor Copiktra (duvelisib), which won approval in September 2018 as a third-line treatment for relapsed or refractory CLL or SLL, but updated pivotal trial results raised safety questions.

In addition to the serious and fatal toxicities of duvelisib, FDA speakers at the ODAC meeting pointed to an evolved treatment landscape for CLL and SLL, with targeted BTK or BCL2 inhibitors (front-line or second-line), and data pointing to a “potential detriment” in overall survival for duvelisib. But some ODAC members noted that the detriment was likely small and that there is some efficacy even as the data are difficult to interpret.

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Klick Health co-founder and CEO Leerom Segal sat down for a one-on-one with former President Barack Obama in New York on Thursday night for a wide-ranging discussion about his time in office and current political divisiveness in the US, but also on some health and science topics.

Segal, for instance, asked the former chief executive about the decline of trust in science — that in 2022 “vaccines are still controversial and maybe even political”— and how it can be reinstated.

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Bioscience & Technology Business Center The University of Kansas Lawrence, Kansas

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